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31 Study Matches

Cladribine Tablets After Treatment With Natalizumab (CLADRINA)

The purpose of this study is to generate hypotheses regarding the safety, efficacy, and immunological impact of cladribine tablets after treatment with natalizumab in patients with relapsing-remitting multiple sclerosis (RRMS) and active secondary progressive multiple sclerosis (active SPMS).
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studyfinder@utsouthwestern.edu
Olaf Stuve
58631
All
18 Years to 60 Years old
Phase 4
This study is NOT accepting healthy volunteers
NCT04178005
STU-2019-1618
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Inclusion Criteria:
Patients who meet the following inclusion criteria will be eligible for enrollment in the study: 1. Age between 18 and 60 years, inclusive. 2. Diagnosis of relapsing forms of MS, to include RRMS and active SPMS, diagnosed with McDonald Criteria 2005, 2010, and/or 2017 (1-3) 3. EDSS 0
•5.5 (Functional system changes in cerebral (or mental) functions and in bowel and bladder functions not used in determining EDSS for protocol eligibility). 4. Has had a minimum of 12 months of continuous natalizumab therapy (300 mg/d), including patients receiving extended interval dosing of natalizumab (e.g., less frequently than every-4-week infusion). 5. Negative history for any relapses at least 28 days prior to enrollment. 6. Weighing between 40 kilograms or more. 7. Female subjects of childbearing potential must use effective methods of contraception to prevent pregnancy for 4 weeks before initiation of cladribine tablets and must agree to continue to practice adequate contraception for at least 6 months after the last dose. Women using systemically acting hormonal contraceptives should add a barrier method during cladribine treatment and for at least 4 weeks after the last dose in each treatment year. 8. Female subjects must not be pregnant; female subjects must not be lactating or breast-feeding at least 10 days after the last dose. 9. Male subjects must be willing to use a condom during dosing and for six months after the last dose. Alternatively, their female partner must use another form of contraception (such as an intra-uterine device [IUD], barrier method with spermicide, or hormonal contraceptive [e.g., implant, injectable, patch or oral]) during dosing and for six months after last dose. 10. Understands and is capable of following through with study protocol requirements and assessments. 11. Willing to provide voluntary and informed consent based on the Health Insurance Portability and Accountability Act (HIPPA).
Exclusion Criteria:
Patients who meet any of the following exclusion criteria will not be eligible for enrollment in the study: 1. Natalizumab failure based on clinician's discretion. 2. Not active progressive MS (4). 3. A diagnosis of PML or any suspicion of PML. 4. A diagnosis of Clinically Isolated Syndrome 5. Known hypersensitivity to cladribine. 6. Any prior exposure to cladribine. 7. Lymphocyte count not within normal limits of the local, hospital laboratory. 8. Previous or current exposure to mitoxantrone, azathioprine, methotrexate, cyclophosphamide, myelosuppressive treatments, total lymphoid irradiation. 9. Receiving oral or systemic corticosteroid treatments within the 28 days prior to enrollment. 10. Receiving cytokine base treatment, Intra Venous Immuno Globulin (IVIG) or Plasma pheresis, 3 months prior to enrollment in the study. 11. Having platelet count or neutrophil count below the lower limit of the normal range within the 28 days prior to enrollment in the study. 12. Positive for HIV, or positive hepatitis C antibody test or hepatitis B surface antigen test and/or core antibody test for IgG and/or IgM. 13. History of tuberculosis (TB), presence of active tuberculosis, or latent tuberculosis as detected by local standard of practice like imaging (e.g., chest X-ray, chest CT scan, MRI) and/or positive QuantiFERON-TB Gold test and/or skin test and/or clinical examination or has had latent TB disease at any time in the past. 14. Immunocompromised subjects, including subjects currently receiving immunosuppressive or myelosuppressive therapy with, e.g., monoclonal antibodies, methotrexate, cyclophosphamide, cyclosporine or azathioprine, or chronic use of corticosteroids. 15. Active malignancy or history of malignancy. 16. Received a live vaccine within 6 weeks prior to cladribine tablet administration or intends to receive a live vaccination during the trial. After the last dose of cladribine tablets, the subject should avoid live vaccine as long as the subject's white blood cell counts are not within normal limits. 17. Allergy or hypersensitivity to gadolinium and/or any other contraindication to perform an MRI. 18. Has any renal condition that would preclude the administration of gadolinium (e.g. acute or chronic severe renal insufficiency (GFR < 30 mL/min/1.73m2) -
Drug: Cladribine
Multiple Sclerosis
UT Southwestern
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A Study of LY3471851 in Adults With Systemic Lupus Erythematosus (SLE) (ISLAND-SLE)

The reason for this study is to see if the study drug LY3471851 (NKTR-358) is safe and effective in adults with systemic lupus erythematosus (SLE).
Call 214-648-5005
studyfinder@utsouthwestern.edu
David Karp
13762
All
18 Years to 65 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04433585
STU-2020-0756
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Inclusion Criteria:

• Have a clinical diagnosis of SLE at least 24 weeks prior to screening.
• Have documentation of having met at least 4 of 11 Revised Criteria for Classification of Systemic Lupus Erythematosus according to the 1997 Update of the 1982 American College of Rheumatology (ACR) criteria for classification of SLE prior to randomization.
• Have a positive antinuclear antibody (ANA) (titer ≥1:80) and/or a positive anti-double-stranded deoxyribonucleic acid (dsDNA), and/or a positive anti-Smith (anti-Sm) as assessed by a central laboratory during screening.
• Have a total Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score ≥6 during screening.
• Have a clinical SLEDAI-2K score ≥4 at randomization.
• Have active arthritis and/or active rash.
Exclusion Criteria:

• Have severe active lupus nephritis.
• Have active central nervous system (CNS) lupus.
• Have a history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, hematological, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data.
• Have a current or recent clinically serious viral, bacterial, fungal, or parasitic infection.
Drug: LY3471851, Drug: Placebo
Systemic Lupus Erythematosus
T regulatory cells (Tregs), Interleukin 2, Interleukin-2
UT Southwestern; Parkland Health & Hospital System
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Follow-up of the EPITOPE Study to Evaluate Long-term Efficacy and Safety of DBV712 in Young Children (EPOPEX)

Open-label, follow-up study for subjects who completed the EPITOPE study.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Christopher Parrish
168280
All
2 Years to 4 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03859700
STU-2019-1180
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Inclusion Criteria:

• completion of the EPITOPE study
Exclusion Criteria:

• Generalized dermatologic disease (for example, active atopic dermatitis, uncontrolled generalized active eczema, ichthyosis vulgaris) extending widely on the skin and especially on the back or arms with no intact zones to apply the Viaskin patches.
• Diagnosis of asthma that evolved to severe, unstable or uncontrolled asthma
Biological: DBV712 250 mcg
Peanut Allergy
EPIT, Epicutaneous, Immunotherapy, Viaskin
Children’s Health
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Vagus Nerve Stimulation for Moderate to Severe Rheumatoid Arthritis (RESET-RA)

The RESET-RA study will assess the safety and efficacy of the SetPoint System (study device) for the treatment of adult patients with active, moderate to severe rheumatoid arthritis who have had an inadequate response or intolerance to biologic or targeted synthetic Disease-Modifying Anti-Rheumatic Drugs (DMARDs). The study device contains a miniaturized stimulator (implant) that is surgically placed under general anesthesia on the vagus nerve through a small incision on the left side of the neck (implant procedure). The study will enroll 250 subjects at 40 sites. All eligible subjects will undergo the implant procedure. Half of the subjects will receive active stimulation (treatment) and the other half will receive non-active stimulation (control). After completing primary endpoint assessments at Week 12, there will be a one-way crossover of control subjects to active stimulation and a 180-week open-label follow-up with all subjects (treatment and control) receiving active stimulation to evaluate long-term safety.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Bradley Lega
153415
All
22 Years to 75 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04539964
STU-2020-1294
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Inclusion Criteria:

• 22-75 years of age at screening
• Active moderate or severe RA, defined as at least 4/28 tender and 4/28 swollen joints
• Demonstrated an inadequate response, loss of response, or intolerance to 1 or more approved for rheumatoid arthritis biologic or targeted synthetic Disease-Modifying Anti-Rheumatic Drugs (DMARDs), including Janus kinase inhibitors (JAKi)
• Receiving treatment with at least 1 conventional synthetic DMARD for at least 12 weeks and on a continuous non-changing dose and route of administration for at least 4 weeks prior to Screening and able to continue the same stable dose through Week 12
Exclusion Criteria:

• Untreated or poorly controlled psychiatric illness or history of substance abuse
• Significant immunodeficiency due to underlying illness
• History of stroke or transient ischemic attack, or diagnosis of cerebrovascular fibromuscular dysplasia
• Clinically significant cardiovascular disease
• Neurological syndromes, including multiple sclerosis, Alzheimer's disease, or Parkinson's disease
• Uncontrolled fibromyalgia
• History of left or right carotid surgery
• History of unilateral or bilateral vagotomy, partial or complete splenectomy
• Recurrent vasovagal syncope episodes
• Current, regular use of tobacco products
• Hypersensitivity/allergy to MRI contrast agents and/or unable to perform MRI
Procedure: Implant Procedure, Drug: Conventional Synthetic DMARD, Device: Active stimulation, Device: Non-active stimulation
Rheumatoid Arthritis
Rheumatoid Arthritis, Vagus nerve, vagus nerve stimulating device, drug refractory, permanent implantable, implant
UT Southwestern
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Study to Investigate the Safety of the Transplantation of Human Glial Restricted Progenitor Cells Into Subjects With Transverse Myelitis

This study is a non-randomized, open-label, partially blinded, sequential cohort, dose-escalation study designed to obtain preliminary data on the safety, tolerability, and early activity of Q-Cells® transplantation in subjects with Transverse Myelitis. For each of the dose levels, transplantation of Q-Cells® unilaterally into spinal cord demyelinated lesions will be evaluated. Subjects will be blinded to side of treatment. Idiopathic Transverse Myelitis is a monophasic disorder characterized predominantly by demyelination. Patients are left with disability from damage to ascending and descending white matter tracts. Q-Cells® are comprised of glial progenitor cells.It is postulated that the Q-Cells® glial progeny (healthy astrocytes and oligodendrocytes) will integrate into the spinal cord lesion site and remyelinate demyelinated axons as well as provide trophic support for damaged axons. Therefore, Q-Cells® have the potential to repair damage that has occurred and could be clinically useful for patients with disability caused by TM. The study is planned to enroll up to 9 subjects. Each subject will be followed for 9 months after transplantation of Q-Cells®. Each subject will receive a single time point administration of Q-Cells®: with transplantation foci targeted to posterior columns in the spinal cord (all transplantation foci below C7) on one side. Study participation consists of Screening, Pre-operative/Treatment, and Post-treatment study periods that will generally last from 9 to 12 months in total. The study data will be assessed for safety and activity until the last subject has completed the 9-month study visit. Following completion of the 9-month follow-up period, subjects who consent will continue to be followed for safety and activity in a separate long-term follow-up protocol.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Benjamin Greenberg
105091
All
18 Years to 70 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT03887273
STU 052017-076
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Inclusion Criteria:
1. Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to collect and use protected health information (PHI) in accordance with national and local subject privacy regulations. 2. Live within reasonable travel distance to center or have reliable mechanism to travel to the center. 3. Have a caregiver willing/able to assist in the transportation and care required by study participation. 4. Subject is 18
•70 years of age (inclusive) on day of Screening Visit. 5. Subject is diagnosed with idiopathic TM within the past 120 months in accord with the Transverse Myelitis Consortium Working Group (2002). 6. Subject has a MRI with a single focus of T2 hyperintensity that is 4 to 10 cm in length if no post contrast enhancement seen, or a single focus T1 post contrast enhancing lesion of 4 to 10 cm, with its most rostral extent at or below C8 myotome/dermatome level. 7. Subject has negative NMO IgG (anti-AQP4) test at two separate time points, separated by at least 6 months. 8. Subject has brain MRI not consistent with multiple sclerosis or other autoimmune or demyelinating disease. 9. Subject is more than 12 months from TM onset. 10. Subject has ASIA A categorization. 11. Subject's neurological deficits related to TM have been stable for at least 3 months. 12. Subject is medically able to undergo the study procedures and physically able to adhere to the visit schedule at the time of study entry. 13. For women of child bearing capacity, negative pregnancy test during the Screening Period and at the Pre-Operative Visit. 14. Males and females will agree to practice effective birth control during study participation and up to one year after.
Exclusion Criteria:
1. Subject with causes of weakness, sensory loss and/or autonomic dysfunction other than TM have not been practically excluded. 2. Subject with significant cognitive impairment, clinical dementia, or major psychiatric illness including psychosis, bipolar disease, major depression, as determined by the DSM-V. 3. Subject with a diagnosis of a neurodegenerative disease (e.g., ALS, Parkinson's disease, Alzheimer's disease). 4. Subject suffering with medical conditions that impair nerve or muscle function (e.g., notable peripheral neuropathy, metabolic muscle disease) or any disease or condition that would impair the subject's neuromuscular function or impair the adequate assessment of the subject's function (e.g., severe osteoarthritis). 5. Subject with a clinically significant history of unstable cardiac, pulmonary, renal, hepatic, endocrine, hematologic, or active malignancy or infectious disease or other medically significant illness that may render them at an unacceptable risk for surgery or that may cause them to be unable to complete the scheduled duration of the trial. 6. History of spine surgery or anatomic variation incompatible with route of administration (as determined by neurosurgeon). 7. Severe spinal stenosis or cord compression causing myelopathy. 8. Abnormal flow voids on the surface of the spinal cord suggestive of arteriovenous malformation (AVM) or evidence of a vascular cause of a myelopathy (e.g., infarct of spinal artery). 9. Any evidence of CNS malignancy or clinically significant CNS lesions as defined by imaging studies of the CNS (MRI of brain and spinal cord). 10. Uncontrolled hypertension (Systolic BP>180mmHg and/or Diastolic BP >110mmHg). 11. Any history of thrombotic or embolic events. 12. Any poorly controlled medical conditions that, in the opinion of the site investigator and/or surgeon, increase risk of surgery to a medically unacceptable degree. 13. Subjects who cannot undergo MRI examination because of any contraindication to the procedure, including the presence of a pacemaker, an implanted defibrillator or certain other implanted electronic or metallic devices, or who have been or might have been exposed to metal fragments, or any reason the subject cannot undergo an MRI routinely for the duration of the trial. 14. Subject with clinically significant abnormal clinical laboratory values, as determined by the Investigator at the screening visit (Visit 1). 15. Subject who is immune compromised (by therapeutic agent or disease) or who has a condition contraindicated to treatment with immunosuppression agents (e.g., tuberculosis, latent infection) as determined by history or testing. Any subject with an ongoing infection until it has been adequately treated and it is deemed to be resolved. 16. Subject with aspartate aminotransferase (AST) or alanine aminotransferase (ALT) value >3.0 times the upper limit of normal at the screening visit (Visit 1). 17. Subject with diabetes or HgbA1c > 6.5 18. Subject with a history of alcohol or drug abuse or dependence within 1 year of screening visit (Visit 1), per DSM-V criteria. 19. Subject unlikely to comply with study requirements, as determined by Investigator. 20. Subject who has been exposed to any other experimental agent (off-label use or investigational) within 60 days of screening visit (Visit 1). Biologic agents may need additional time for washout and will be evaluated by the Sponsor on a case-by-case basis. 21. Subject with pre-existing anti-human leukocyte antigen (HLA) class I or class II antibodies directed against the Q-Cells®, as determined by panel reactive antibody (PRA) assay. 22. Allergy to study treatment or any of its constituents (e.g., chicken eggs), or allergy to any of the co-administered immunosuppressants or any of their excipients. 23. Subject with any medical condition or using concomitant medication that would contraindicate the use of tacrolimus, mycophenolate mofetil, or prednisone as determined by Investigator. 24. Subject has undergone stem cell transplantation (including T-cell or bone marrow transplants) at any time prior to study (within or outside the US). 25. Subject with evidence of deep vein thrombosis (DVT) by venous ultrasound or any previous evidence of DVT. 26. Subject has recent (1 year) or recurrent history of gastrointestinal bleeding or peptic ulcer disease or is under active treatment to prevent recurrence. 27. Subject with estimated glomerular filtration rate at screening of less than 60 mL/min/1.73m2. 28. Subjects with hereditary deficiency of hypoxanthine-guanine phosphoribosyl-transferase (HGPRT) such as Lesch-Nyhan and Kelley-Seegmiller syndrome. 29. Vaccination with live virus within 6 weeks of screening. 30. History or evidence of optic neuritis. 31. Any reason, in the judgment of the investigator, which would make the subject inappropriate for entry into this trial.
Biological: Q-Cells
Transverse Myelitis, Brain and Nervous System
UT Southwestern
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Study of Anti-Malarials in Incomplete Lupus Erythematosus (SMILE)

This project is a multicenter, randomized, placebo-controlled, double-blind clinical trial that is designed to test whether treating patients who are at risk for development of lupus with hydroxychloroquine can slow accumulation of disease features. Effects on clinical progression of symptoms, patient-reported outcomes and changes in the immune markers of response will be measured and toxicity of the treatment will be assessed. This trial is a first step in testing a prevention strategy for lupus.
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David Karp
13762
All
15 Years to 49 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03030118
STU 112015-016
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Inclusion Criteria:
1. Between 15 and 49 years of age, inclusive, at Visit 1. 2. Anti-nuclear antibody (ANA) titer of 1:80, or greater, as determined by immunofluorescence assay (IFA). 3. Participants must have at least one (but not three or more) additional clinical or laboratory criterion from the 2012 Systemic Lupus International Collaborating Clinics (SLICC) classification criteria. 4. Written informed consent (and assent when applicable) obtained from subject or subject's legal representative and ability for subject to comply with the requirements of the study.
Exclusion Criteria:
1. The subject meets the 2012 SLICC classification criteria for SLE at Visit 1 (i.e., ANA plus 3 other criteria, or ANA plus biopsy-proven lupus nephritis). 2. The subject has been diagnosed with another autoimmune disorder, other than autoimmune thyroid conditions. 3. The subject has fibromyalgia, based on clinical history and exam. 4. The subject has previously been or is currently being treated with oral antimalarial agents including hydroxychloroquine, chloroquine, or quinacrine. 5. The subject is currently or has been treated with immunosuppressive, immune modifying, or cytotoxic medications as listed in Section 7.2. 6. Use of any investigational agent within the preceding 12 months. 7. History of primary immunodeficiency. 8. Active bacterial, viral, fungal, or opportunistic infection. 9. Evidence of infection with human immunodeficiency virus (HIV), Hepatitis B, or Hepatitis C. 10. Concomitant malignancy or history of malignancy with the exception of adequately treated basal or squamous cell carcinoma of the skin, or carcinoma in situ of the cervix. 11. The subject has significant findings on ophthalmological examination that, in the opinion of the examining Ophthalmologist, prevent safe use of hydroxychloroquine. 12. The subject has other contraindications to treatment with hydroxychloroquine including pre-existing ocular disease, hepatic impairment, psoriasis, porphyria, or allergy to the drug or class. 13. Co-morbidities requiring systemic corticosteroid therapy greater than 10 mg of prednisone per day, or equivalent, or a change in corticosteroid dose within the 3 months prior to Visit 1. 14. Starting, stopping, or changing the dose of over the counter or prescription non-steroidal anti-inflammatory drugs (NSAIDs) in the three months prior to Visit 1. 15. Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study. 16. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data. 17. Inability to comply with the study visit schedule and procedures.
Drug: Hydroxychloroquine, Drug: Placebo Oral Capsule
Systemic Lupus Erythematosus
hydroxychloroquine
UT Southwestern; Parkland Health & Hospital System
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A Study To Evaluate The Efficacy And Safety Of Obinutuzumab In Patients With ISN/RPS 2003 Class III Or IV Lupus Nephritis (REGENCY)

This study will evaluate the efficacy, safety, and pharmacokinetics of obinutuzumab compared with placebo in patients with International Society of Nephrology/Renal Pathology Society (ISN/RPS) class III or IV lupus nephritis (LN) when added on to standard-of-care therapy consisting of mycophenolate mofetil (MMF) and corticosteroids.
Call 214-648-5005
studyfinder@utsouthwestern.edu
David Karp
13762
All
18 Years to 75 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04221477
STU-2020-0374
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Key
Inclusion Criteria:

• Diagnosis of ISN/RPS 2003 Class III or IV LN as evidenced by renal biopsy performed within 6 months. Participants may co-exhibit Class V disease in addition to either Class III or Class IV disease
• Urine protein to creatinine ratio greater than or equal to (>/=) 1 on a 24-hour collection
• Other inclusion criteria may apply Key
Exclusion Criteria:

• Pregnancy or breastfeeding
• Severe renal impairment or the need for dialysis or renal transplantation
• Receipt of an excluded therapy, including any anti-CD20 therapy less than 9 months prior to screening or during screening; or cyclophosphamide, tacrolimus, ciclosporin, or voclosporin during the 2 months prior to screening or during screening
• Significant or uncontrolled medical disease which, in the investigator's opinion, would preclude patient participation
• Known active infection of any kind or recent major episode of infection
• Intolerance or contraindication to study therapies
• Other exclusion criteria may apply
Drug: Obinutuzumab, Drug: MMF, Drug: Prednisone, Drug: Placebo, Drug: Methylprednisolone, Drug: Acetaminophen, Drug: Diphenhydramine
Lupus Nephritis
UT Southwestern
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Long-term Safety Study of AR101 in Subjects Who Participated in a Prior AR101 Study (ARC008)

The purpose of this study is to assess AR101's safety and tolerability over an extended dosing period.
Call 214-648-5005
studyfinder@utsouthwestern.edu
John Bird
108478
All
4 Years to 55 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03292484
STU 092017-039
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Key
Inclusion Criteria:

• Prior participation in an Aimmune AR101 clinical study or any future clinical study that identifies ARC008 as a follow-on study option in the protocol
• Written informed consent and/or assent from subjects/guardians as appropriate
• Use of effective birth control by sexually active female subjects of childbearing potential Key
Exclusion Criteria:

• Did not complete a minimum of 3 months of AR101 maintenance therapy if the subject was assigned to AR101 in the parent study
• Early discontinuation from the parent study
• In subjects treated with AR101 in the parent study requiring a food challenge, failure to successfully consume at least 300 mg single dose of peanut protein at parent study's exit food challenge
Biological: AR101
Peanut Allergy, Other
AR101, Characterized Peanut Allergen, OIT (oral immunotherapy), Peanut Allergy, Allergy, Peanut-Allergic Children, Peanut-Allergic Adults, Desensitization, CPNA (Characterized Peanut Allergen)
Children’s Health
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STeroids to REduce Systemic Inflammation After Infant Heart Surgery (STRESS)

This study's objective is to determine the pharmacokinetics (PK)/pharmacodynamics (PD), safety and efficacy of methylprednisolone in infants undergoing heart surgery with cardiopulmonary bypass. This is a prospective, double blind, multi-center, placebo-controlled safety and efficacy study. Blood samples will be collected from a subset of enrolled study participants to evaluate multiple dose methylprednisolone PK/PD. Participants will be randomized in a 1:1 fashion to intravenous methylprednisolone versus placebo. Study drug/placebo will be administered 8 to 12 hours before the anticipated start time of surgery and in the operating room at the time of initiation of cardiopulmonary bypass. Patients will be followed for primary and secondary outcomes for the duration of their hospitalization. Serious study drug-related adverse events will be collected for 7 days after the last dose of study drug.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Ryan Butts
169606
All
up to 12 Months old
Phase 3
This study is NOT accepting healthy volunteers
NCT03229538
STU 072017-052
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Inclusion Criteria:

• Age < 1 year at the time of surgery
• Undergoing heart surgery with CPB as part of standard clinical care
• Availability and willingness of the parent/legally authorized representative to provide written informed consent
Exclusion Criteria:

• < 37 weeks adjusted gestational age at time of surgery
• Any oral or intravenous steroid treatment within two days of surgery
• Any patient receiving any of the following medications within 2 days of surgery: Amphoteracin B, aminoglutethimide, anticholesterases, warfarin, P450 3A4 inducers including (but not limited to) carbamazepine, phenobarbital, phenytoin, rifampin, bosentan and nafcillin or P450 3A4 inhibitors including (but not limited to) clarithromycin, voriconazole, itraconazole, ketoconazole, ciprofloxacin, diltiazem, fluconazole, erythromycin and verapamil.
• Infection contraindicating steroid use
• Preoperative mechanical circulatory support or active resuscitation at the time of randomization
• Emergent surgery precluding steroid administration 8-12 hours before surgery
Drug: Methylprednisolone, Drug: Isotonic saline
Congenital Heart Disease in Children, Inflammatory Response, Cardiovascular
Children’s Health
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Tacrolimus/Everolimus vs. Tacrolimus/MMF in Pediatric Heart Transplant Recipients Using the MATE Score (TEAMMATE)

The TEAMMATE Trial will enroll 210 pediatric heart transplant patients from 25 centers at 6 months post-transplant and follow each patient for 2.5 years. Half of the participants will receive everolimus and low-dose tacrolimus and the other half will receive tacrolimus and mycophenolate mofetil. The trial will determine which treatment is better at reducing the cumulative risk of coronary artery vasculopathy, chronic kidney disease and biopsy proven-acute cellular rejection without an increase in graft loss due to all causes (e.g. infection, PTLD, antibody mediated rejection).
Call 214-648-5005
studyfinder@utsouthwestern.edu
Ryan Butts
169606
All
up to 21 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03386539
STU 122017-025
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Inclusion Criteria:
1. Orthotopic heart transplantation 2. Age < 21 years at time of transplant 3. Stable immunosuppression at the time of randomization with no contraindication to everolimus, tacrolimus, or mycophenolate mofetil 4. Planned follow-up at a study site for the 30 month duration of the study. 5. Subject or legal adult representative capable of providing informed consent (in general, assent will be sought for children aged 12 years or older).
Exclusion Criteria:
1. Multi-organ transplant (e.g. heart-lung or heart-liver). 2. Known hypersensitivity to everolimus, sirolimus, tacrolimus or mycophenolate mofetil (MMF), or to components of the drug products. 3. Patients on maintenance corticosteroid therapy exceeding a dose equivalent of prednisone 0.1 mg/kg/day at randomization. 4. High-risk for rejection defined as active rejection, recurrent (≥ 2 episodes of grade 2R rejection) cellular rejection, recurrent rejection (≥ 2 episodes of any grade) with hemodynamic compromise, steroid-resistant rejection or unresolved antibody-mediated rejection during the first 6 months post-heart transplant 5. Graft dysfunction (LVEF <40% or wedge pressure >22 mmHg or cardiac index <2.2 L/min/m2) 6. Stage 4 or 5 CKD (eGFR <30 ml/min/1.73 m2) or moderate proteinuria (urine protein to urine creatinine ratio >0.5 mg/mg). 7. Active infection requiring hospitalization or treatment dose medical therapy. 8. Patients with ongoing wound healing problems, clinically significant wound infection requiring continued therapy or other severe surgical complication in the opinion of the Site Principal Investigator. 9. Fasting Serum Cholesterol ≥300 mg/dL OR greater than or equal to 7.75 mmol/L, AND fasting triglycerides ≥2.5x the upper limit of normal (ULN). Note: In case one or both of these thresholds are exceeded, the patient can only be included after initiation of appropriate lipid lowering medication, and reduction of serum cholesterol and triglyceride levels to below exclusion ranges is confirmed. 10. Uncontrolled diabetes mellitus. 11. Diagnosis of post-transplant lymphoproliferative disorder (PTLD) during the first 6 months post-heart transplant. 12. History of non-adherence to medical regimens. 13. Patients who are treated with drugs that are strong inducers or inhibitors of cytochrome P450 3A4 (CYP3A4) and cannot discontinue the treatment 14. Patients who are pregnant or breast-feeding or intend to get pregnant during the study period.
Drug: Everolimus, Drug: Tacrolimus, Drug: Mycophenolate Mofetil
Post-transplant Lymphoproliferative Disorder, Chronic Kidney Diseases, Pediatric Heart Transplantation, Immunosuppression, Cardiac Allograft Vasculopathy, Heart Transplant Failure and Rejection, Heart Transplant Infection
heart transplantation, children, everolimus, tacrolimus, mycophenolate mofetil, randomized clinical trial
Children’s Health
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Omalizumab as Monotherapy and as Adjunct Therapy to Multi-Allergen OIT in Food Allergic Participants (OUtMATCH)

This study is a multi-center, randomized, double-blind, placebo-controlled study in participants 1 to less than 56 years of age who are allergic to peanut and at least two other foods (including milk, egg, wheat, cashew, hazelnut, or walnut). While each participant may be allergic to more than two other foods, the primary endpoint/outcome in this study will only be assessed in peanut and two other foods for each participant. The primary objective of the study is to compare the ability to consume foods without dose-limiting symptoms during a double-blind placebo-controlled food challenge (DBPCFC), after treatment with either omalizumab or placebo for omalizumab.
Call 214-648-5005
studyfinder@utsouthwestern.edu
John Bird
108478
All
1 Year to 55 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03881696
STU-2019-0579
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Inclusion Criteria:
Individuals who meet all of the following criteria are eligible for enrollment as study participants: 1. Participant and/or parent/legal guardian must be able to understand and provide informed consent and/or assent, as applicable; 2. Peanut allergic: participant must meet all of the following criteria to minimize the chance that the participant will develop natural tolerance to peanut over the course of the study: 1. Positive skin prick test (SPT) defined as ≥4 mm wheal greater than saline control) to peanut, 2. Positive peanut immunoglobulin E (IgE), ≥6 kUA/L, at Screening or within three months of Screening, determined by ImmunoCap, and 3. Positive double-blind placebo-controlled food challenge (DBPCFC), defined as experiencing dose-limiting symptoms at a single dose of ≤100 mg of peanut protein. 3. Allergic to at least two of the six other foods (milk, egg, wheat, cashew, hazelnut, walnut): each participant must meet all of the following criteria for at least two of the six other foods to minimize the chance that the participant will develop natural tolerance to at least two of the six other foods over the course of the study: 1. Positive SPT (≥4 mm wheal) to food, 2. Positive food specific IgE (≥6 kUA/L) at Screening or within three months of Screening, determined by ImmunoCap, and 3. Positive DBPCFC, defined as experiencing dose-limiting symptoms at a single dose of ≤300 mg of food protein. 4. With body weight (as measured at Screening) and total serum IgE level (as measured within three months of Screening) suitable for omalizumab dosing; 5. If female of child-bearing potential, must have a negative urine or serum pregnancy test; 6. For women of childbearing potential, must agree to,during the treatment period and for 60 days after the last dose of study drug:
• remain abstinent (refrain from heterosexual intercourse), or
• use acceptable contraceptive methods (barrier methods, or
• oral, injected, or implanted hormonal methods of contraception, or
• other forms of hormonal contraception that have comparable efficacy). 7. Plan to remain in the study area of an OUtMATCH clinical research unit (CRU) during the trial; and 8. Be willing to be trained on the proper use of an epinephrine autoinjector for the duration of the study.
Exclusion Criteria:
Individuals who meet any of the following criteria are not eligible for enrollment as study participants: 1. Inability or unwillingness of a participant and/or parent/legal guardian to give written informed consent and/or assent or comply with the study protocol; 2. Clinically significant laboratory abnormalities at Screening; 3. Dose-limiting symptoms to the placebo portion of the Screening DBPCFC; 4. Sensitivity or suspected/known allergy to any ingredients (including excipients) of the
• active or placebo oral food challenge (OFC) material,
• multi-allergen oral immunotherapy (OIT), or
• drugs related to omalizumab (e.g., monoclonal antibodies, polyclonal gamma globulin).
• Note: Guidance for determination of sensitivity to excipients will be detailed in the study's Manual of Procedures (MOP). 5. Poorly controlled atopic dermatitis (AD) at Screening, per the Principal Investigator's PI's) discretion; 6. Poorly controlled or severe asthma/wheezing at Screening, defined by at least one of the following criteria: 1. Global Initiative for Asthma (GINA) criteria regarding asthma control latest guidelines, 2. History of two or more systemic corticosteroid courses within six months of Screening or one course of systemic corticosteroids within three months of Screening to treat asthma/wheezing, 3. Prior intubation/mechanical ventilation for asthma/wheezing, 4. One hospitalization or Emergency Department (ED) visit for asthma/wheezing within six months of Screening, 5. Forced expiratory volume in one second (FEV1) <80 percent of predicted or FEV1/forced vital capacity (FVC) <75 percent, with or without controller medications (only for participants who are aged seven years or older and are able to perform spirometry), or 6. Inhaled corticosteroid (ICS) dosing of >500 mcg daily fluticasone (or equivalent ICS based on the National Institutes of Health, National Heart, Lung, and Blood Institute (NHLBI) dosing chart). 7. History of severe anaphylaxis to participant-specific foods that will be used in this study, defined as neurological compromise or requiring intubation; 8. Treatment with a burst of oral, intramuscular (IM), or intravenous (IV) steroids of more than two days for an indication other than asthma/wheezing within 30 days of Screening; 9. Currently receiving oral, intramuscular, or intravenous corticosteroids, tricyclic antidepressants, or beta-blockers (oral or topical); 10. Past or current history of eosinophilic gastrointestinal (GI) disease within three years of Screening; 11. Past or current history of cancer, or currently being investigated for possible cancer; 12. Previous adverse reaction to omalizumab; 13. Past or current history of any immunotherapy to any of the foods being treated in this study (e.g., OIT, sublingual immunotherapy [SLIT], EPIT) within 6 months of Screening; 14. Treatment with monoclonal antibody therapy, such as omalizumab (Xolair®), dupilumab (Dupixent®), benralizumab (Fasenra™), mepolizumab (Nucala®), reslizumab (Cinqair®), or other immunomodulatory therapy within six months of Screening; 15. Currently on "build-up phase" of inhalant allergen immunotherapy (i.e., has not reached maintenance dosing). Note: Individuals tolerating maintenance allergen immunotherapy can be enrolled; 16. Inability to discontinue antihistamines for the minimum wash-out periods required for SPTs,or OFCs; 17. Current participation in another therapeutic or interventional clinical trial or participation within 90 days of Screening; 18. Use of investigational drugs within 24 weeks of Screening; 19. Pregnant or breastfeeding, or intending to become pregnant during the study or within 60 days after the last dose of omalizumab or placebo for omalizumab; 20. Has a first-degree relative already enrolled in the study; or 21. Past or current medical problems (e.g., severe latex allergy), history of other chronic diseases (other than asthma/wheezing, AD, or rhinitis) requiring therapy (e.g., heart disease, diabetes), findings from physical assessment, or abnormalities in clinical laboratory testing that are not listed above, which, in the opinion of the PI, may:
• pose additional risks from participation in the study,
• may interfere with the participant's ability to comply with study requirements, or
• may impact the quality or interpretation of the data obtained from the study.
Drug: Omalizumab, Drug: Placebo for Omalizumab, Drug: Multi-Allergen Oral Immunotherapy, Drug: Placebo for Multi-Allergen Oral Immunotherapy, Other: Double-Blind Placebo-Controlled Food Challenge Based Treatment
Peanut Allergy, Multi-food Allergy, Other
peanut allergic, multi-food allergic, omalizumab, placebo, multi-allergen oral immunotherapy (OIT), double-blind placebo-controlled food challenge (DBPCFC), oral food challenge (OFC)
Children’s Health
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A Study to Evaluate the Efficacy and Safety of Dapirolizumab Pegol in Study Participants With Moderately to Severely Active Systemic Lupus Erythematosus (PHOENYCS GO)

The purpose of this study is to evaluate the ability of dapirolizumab pegol (DZP) as an add-on treatment to standard of care (SOC) medication to achieve clinically relevant long term improvement of moderate to severe disease activity.
Call 214-648-5005
studyfinder@utsouthwestern.edu
David Karp
13762
All
16 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04294667
STU-2020-0695
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Inclusion Criteria:

• Study participant must be ≥16 years of age
• Study participants who have moderate to severe disease activity due to either persisting active SLE or due to an acute worsening of SLE in the scope of frequent flaring/relapsing-remitting systemic lupus erythematosus (SLE) despite stable standard of care (SOC) medication defined as: 1. Diagnosed with SLE at least 24 weeks before the Screening Visit (Visit 1) study entry by a qualified physician 2. Classified by 2019 SLE European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) classification criteria for SLE 3. With serological evidence for SLE at Screening as demonstrated by at least 1 of the following: i) Evidence for anti-dsDNA (in central laboratory at Screening) ii) Either complement C3 < lower limit of normal (LLN) OR complement C4 • British Isles Lupus Assessment Group Disease Activity Index 2004 (BILAG 2004) Grade B in ≥2 organ systems and/or a BILAG 2004 Grade A in ≥1 organ systems at Screening and Baseline Visit AND
• Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) ≥6 at Screening Visit AND
• SLEDAI-2K without labs ≥4 at Baseline Visit e. Receiving the following SOC medication at stable dose:
• Antimalarial treatment in combination with corticosteroids and/or immunosuppressants or as stand-alone treatment if justified OR Treatment with corticosteroids and/or immunosuppressants if anti-malarial treatment is not possible
Exclusion Criteria:
- Study participant has any medical or psychiatric condition (including conditions due to neuropsychiatric systemic lupus erythematosus (SLE)) that, in the opinion of the Investigator, could jeopardize or would compromise the study participant's ability to participate in this study. This includes study participants with a life threatening condition
• Study participant has a history of an anaphylactic reaction to parenteral administration of contrast agents, human or murine proteins, or monoclonal antibodies
• Study participant has a history of malignancy, except the following treated cancers: cervical carcinoma in situ, basal cell carcinoma, or dermatological squamous cell carcinoma
• Study participant has an increased risk for thromboembolic events due to an ongoing heart disease or due to a medical device, including but not limited to vascular graft, valvular heart disease, atrial fibrillation, or a heart rhythm disorder
• Study participant has evidence of human immunodeficiency virus (HIV) infection, agammaglobulinemias, T-cell deficiencies, or human T-cell lymphotropic virus-1 infection
• Study participant had a reactivated latent or opportunistic infection within 12 weeks prior to the first study medication infusion (Visit 2), or is currently receiving suppressive therapy for an opportunistic infection
• Study participants who have received live/live attenuated vaccines within 6 weeks prior to the first study medication infusion
• Study participant has clinically significant active or latent infection
• Study participant has a mixed connective tissue disease, scleroderma, and/or overlap syndrome of these diseases with SLE
• Study participant takes any protocol defined prohibited concomitant medication
• Study participant has previously been randomized within this study or participant has previously been assigned to treatment with dapirolizumab pegol (DZP) in a study evaluating DZP
• Study participant has participated in another study of an IMP within the previous 12 weeks or 5 half-lives of the investigational medicinal product (IMP) whatever is longer or is currently participating in another study of an IMP
• Study participant has chronic kidney failure stage 4, manifested by estimated glomerular filtration rate <30mL/min/1.73m2, or serum creatinine >2.5 mg/dL, or participant has proteinuria >3 g/day, or protein: creatinine ratio >340 mg/mmol at the Screening Visit
Drug: DZP, Other: Placebo
Systemic Lupus Erythematosus
Systemic lupus erythematosus, Dapirolizumab pegol, SLE, DZP
UT Southwestern; Parkland Health & Hospital System
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A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of a Higher Dose of Ocrelizumab in Adults With Primary Progressive Multiple Sclerosis (PPMS)

This is a randomized, double blind, controlled, parallel group, multicenter study to evaluate efficacy, safety and pharmacokinetics of a higher dose of ocrelizumab per intravenous (IV) infusion every 24 weeks in participants with PPMS, in comparison to the approved 600 mg dose of ocrelizumab.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Peter Sguigna
83433
All
18 Years to 55 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04548999
STU-2021-0017
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Inclusion Criteria:

• Diagnosis of primary progressive multiple sclerosis (PPMS).
• Expanded disability status scale (EDSS) score at screening and baseline, from 3 to 6.5 inclusive.
• Average T25FWT score over two trials at screening and over two trials at baseline respectively, up to 150 (inclusive) seconds
• Average 9HPT score over four trials (two trials with each hand) at screening and over four trials (two trials with each hand) at baseline respectively, up to 250 (inclusive) seconds
• Score of >/= to 2.0 on the Functional Systems scale for the pyramidal system that was due to lower extremity findings at screening and baseline.
• Documented MRI of brain with abnormalities consistent with MS
• Participants requiring symptomatic treatment for MS and/or physiotherapy must be treated at a stable dose. No initiation of symptomatic treatment for MS or physiotherapy within 4 weeks of randomization.
• Participants must be neurologically stable for at least 30 days prior to randomization and baseline.
• Disease duration from the onset of MS symptoms; if EDSS score at screening is less or equal to 5, disease duration must be less than 10 years; If EDSS score at screening is more than 5, disease duration must be less than 15 years
• Documented evidence of the presence of at least one cerebrospinal fluid-specific oligoclonal bands.
• For females of childbearing potential, agreement to remain abstinent or use adequate contraceptive methods.
• For female participants without reproductive potential, may be enrolled if post-menopausal unless receiving a hormonal therapy for her menopause or if surgically sterile.
Exclusion Criteria:

• History of relapsing remitting or secondary progressive MS at screening.
• Any known or suspected active infection at screening or baseline (except nailbed infections), or any major episode of infection requiring hospitalization or treatment with IV antimicrobials within 8 weeks or treatment with oral antimicrobials within 2 weeks, prior to and during screening.
• History of confirmed or suspected progressive multifocal leukoencephalopathy.
• History of cancer, including hematologic malignancy and solid tumors, within 10 years of screening.
• Immunocompromised state.
• Receipt of a live or live-attenuated vaccine within 6 weeks prior to randomization.
• Inability to complete an MRI or contraindication to gadolinium administration.
• Contraindications to mandatory pre-medications for IRRs.
• Known presence of other neurologic disorders that could interfere with the diagnosis of MS or assessments of efficacy and/or safety during the study.
• Any concomitant disease that may require chronic treatment with systemic corticosteroids or immunosuppressants during the course of the study.
• Significant, uncontrolled disease that may preclude participant from participating in the study.
• History of or currently active primary or secondary, non-drug-related, immunodeficiency.
• Pregnant or breastfeeding or intending to become pregnant.
• Lack of peripheral venous access.
• History of alcohol or other drug abuse within 12 months prior to screening.
• Treatment with any investigational agent or treatment with any experimental procedure for MS.
• Previous use of anti-CD20s (including ocrelizumab), unless the last infusion was more than 2 years before screening, B-cell count is normal, and the stop of the treatment was not motivated by safety reasons or lack of efficacy.
• Any previous treatment with mitoxantrone, cladribine, atacicept, alemtuzumab, and daclizumab
• Previous treatment with fingolimod, siponimod, or ozanimod within 6 weeks of baseline
• Previous treatment with natalizumab within 4.5 months of baseline
• Previous treatment with interferons beta (1a or 1b), or glatiramer acetate within 2 weeks of baseline
• Previous treatment with any other immunomodulatory or immunosuppressive medication not already listed above without appropriate washout as described in the applicable local label. If the washout requirements are not described in the applicable local label, then the wash out period must be five times the half-life of the medication.
• Any previous treatment with bone marrow transplantation and hematopoietic stem cell transplantation.
• Any previous history of transplantation or anti-rejection therapy.
• Treatment with intravenous (IV) immunoglobulin (Ig) or plasmapheresis within 12 weeks prior to randomization.
• Systemic corticosteroid therapy within 4 weeks prior to screening.
• Positive screening tests for active, latent, or inadequately treated hepatitis B
• Sensitivity or intolerance to any ingredient (including excipients) of ocrelizumab.
• Any additional exclusionary criterion as per ocrelizumab local label, if more stringent than the above.
Drug: Ocrelizumab, Drug: Ocrelizumab, Drug: Antihistamine, Drug: Methylprednisolone
Multiple Sclerosis
UT Southwestern
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Nivolumab in Treating Patients With Autoimmune Disorders and Advanced, Metastatic, or Unresectable Cancer

This phase Ib trial studies the side effects of nivolumab and to see how well it works in treating patients with autoimmune disorders and cancer that has spread to other places in the body or cannot removed by surgery. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Hans Hammers
169573
All
18 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT03816345
STU-2021-0100
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Inclusion Criteria:

• Patients must have histologically confirmed malignancy that is radiologically evaluable and metastatic or unresectable. Eligible tumor types include solid tumors and malignancies in which there is known evidence of clinical activity for single agent PD-1 or PD-L1 antibodies. Nivolumab is Food and Drug Administration (FDA)-approved for the treatment of melanoma, non-small cell lung cancer (NSCLC), Merkel cell cancer, bladder cancer, renal cell carcinoma (RCC), gastric cancer, hepatocellular carcinoma (HCC), cervical cancer, head and neck cancer, Hodgkin lymphoma (HL), metastatic small cell lung cancer (SCLC), and any solid tumor with microsatellite instability (MSI)-high status confirmed. Patients with HL are eligible but must follow standard response criteria. Additional tumor types may be eligible on a case by case basis upon discussion with principal investigator (PI)
• Patients who have previously received other forms of immunotherapy (high-dose [HD] IL-2, IFN, CTLA-4) are allowed. Patients must not have received cytokine immunotherapy for at least 4 weeks before nivolumab administration. Patients who have received prior anti-CTLA4 will be allowed and the washout period is 6 weeks
• Age >= 18 years; children are excluded from this study but may be eligible for future pediatric phase 1 combination trials
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 (Karnofsky >= 60)
• Life expectancy of greater than 12 weeks
• Leukocytes >= 2,000/mcL
• Absolute neutrophil count >= 1,500/mcL
• Platelets >= 100,000/mcL
• Total bilirubin =< 1.5 x institutional upper limit of normal (ULN)
• Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 2.5 x institutional ULN unless the patient has dermatomyositis and in the opinion of the investigator the elevation is due to diabetes mellitus (DM)
• Creatinine ULN OR glomerular filtration rate (GFR) >= 30 mL/min (if using the Cockcroft-Gault formula)
• Human immunodeficiency virus (HIV)-infected patients on effective antiretroviral therapy with undetectable viral load within 6 months are eligible for this trial
• If evidence of chronic hepatitis B virus (HBV) infection, HBV viral load must be undetectable on suppressive therapy if indicated
• If history of hepatitis C virus (HCV) infection, must be treated with undetectable HCV viral load
• Patients with treated brain metastases are eligible if there is no evidence of progression for at least 4 weeks after central nervous system (CNS)-directed treatment, as ascertained by clinical examination and brain imaging (magnetic resonance imaging [MRI] or computed tomography [CT] scan) during the screening period. Patients with new or progressive brain metastases (active brain metastases) or leptomeningeal disease are eligible if the treating physician determines that immediate CNS specific treatment is not required and is unlikely to be required for at least 4 weeks (or scheduled assessment after the first cycle of treatment), and a risk-benefit analysis (discussion) by the patient and the investigator favors participation in the clinical trial
• The effects of nivolumab on the developing human fetus are unknown. For this reason, women of child-bearing potential (WOCBP) and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. WOCBP receiving nivolumab will be instructed to adhere to contraception for a period of 5 months after the last dose of investigational product. Men receiving nivolumab and who are sexually active with WOCBP will be instructed to adhere to contraception for a period of 7 months after the last dose of investigational product. Women of childbearing potential must have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of human chorionic gonadotropin [HCG]) within 24 hours prior to the start of nivolumab. Women must not be breastfeeding. Women who are not of childbearing potential (i.e., who are postmenopausal or surgically sterile as well as azoospermic men) do not require contraception. WOCBP is defined as any female who has experienced menarche and who has not undergone surgical sterilization (hysterectomy or bilateral oophorectomy) or who is not postmenopausal. Menopause is defined clinically as 12 months of amenorrhea in a woman over 45 in the absence of other biological or physiological causes. In addition, women under the age of 55 must have a documented serum follicle stimulating hormone (FSH) level less than 40 mIU/mL. These durations have been calculated using the upper limit of the half-life for nivolumab (25 days) and are based on the protocol requirement that WOCBP use contraception for 5 half-lives plus 30 days, and men who are sexually active with WOCBP use contraception for 5 half-lives plus 90 days. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she (or the participating partner) should inform the treating physician immediately
• Ability to understand and the willingness to sign a written informed consent document
Exclusion Criteria:

• Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events (AEs) due to agents administered more than 4 weeks earlier have not resolved or stabilized. Palliative (limited-field) radiation therapy (RT) is permitted, if all of the following criteria are met:
• Repeat imaging demonstrates no new sites of bone metastases
• The lesion being considered for palliative radiation is not a target lesion
• Patients with prior therapy with an anti-PD-1 or anti-PD-L1
• Patients with prior allogeneic hematologic transplant
• Patients who are receiving any other investigational agents
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
• Patients who have had evidence of active or acute diverticulitis, intra-abdominal abscess, gastrointestinal (GI) bleeding, obstruction, and abdominal carcinomatosis which are known risk factors for bowel perforation should be evaluated for the potential need for additional treatment before coming on study. For the IBD (UC and CD) cohort, an endoscopic assessment, disease activity index, and disease specific inclusion/exclusion criteria will substitute for these factors in determining eligibility with the exception of abdominal carcinomatosis, which should prompt further evaluation
Biological: Nivolumab
Systemic Lupus Erythematosus, Lymphoma, Sarcoma, Multiple Myeloma, Multiple Sclerosis, Rheumatoid Arthritis, Ulcerative Colitis, Mycosis Fungoides, Dermatomyositis, Advanced Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm, Crohn Disease, Hematopoietic and Lymphoid Cell Neoplasm, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Esophagus, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Thyroid, Urinary Bladder, Leukemia, Other, Hodgkins Lymphoma, Kaposis sarcoma, Leukemia, Not Otherwise Specified, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Non-Hodgkins Lymphoma, Other Hematopoietic, Small Intestine, Soft Tissue, Unknown Sites, Inflammatory Bowel Disease, Autoimmune Disease, Sjogren Syndrome, Systemic Scleroderma, Unresectable Malignant Solid Neoplasm
UT Southwestern
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Assessment of ANK-700 in Patients With Relapsing Remitting Multiple Sclerosis (MoveS-it)

A safety study of ANK-700 in patients with relapsing remitting multiple sclerosis. The study has two parts: Part A - first in human study in which patients receive a single dose of ANK-700 Part B - patients will receive three doses of either ANK-700 or placebo
Call 214-648-5005
studyfinder@utsouthwestern.edu
Benjamin Greenberg
105091
All
18 Years to 55 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT04602390
STU-2020-0880
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Inclusion Criteria:

• Diagnosed with RRMS per revised McDonald criteria (2017) with an EDSS score ≤ 6.5 at screening
• Neurologically stable with no evidence of relapse within the 28 days before signing the informed consent form (ICF)
• Either not currently receiving disease modifying MS therapy, or currently using fumarate drugs (dimethyl fumarate or diroximel fumarate)
• Patients must use a highly effective method of birth control or are sterile or postmenopausal as confirmed by study Investigator
• Patient has signed and understands the ICF
Exclusion Criteria:

• Diagnosis of primary progressive MS or secondary progressive MS
• Uncontrolled or significant medical conditions (including active infection or chronic hepatitis) which, in the opinion of the Investigator, preclude participation
• Patients treated with glatiramer acetate, parenteral steroids or adrenocorticotropic hormone, β-interferon, plasma exchange, fingolimod, ozanimod, or siponimod within the 3 months prior to first dose
• Patients treated with cytotoxic agents (including, but not limited to, cladribine, mitoxantrone, cyclophosphamide, azathioprine, and methotrexate), laquinimod, teriflunomide, or IV gamma globulin within 12 months prior to first dose
• Patients treated with monoclonal antibody therapy (including natalizumab, daclizumab, rituximab, ofatumumab, and ocrelizumab) within 24 months prior to first dose
• Patients previously treated with alemtuzumab, total lymphoid irradiation, mesenchymal stem cell or hematopoietic stem cell transplantation, or tolerance-inducing therapies for MS
• Contraindication to or inability to undergo gadolinium-enhanced magnetic resonance imaging (MRI) scan
• Use of any investigational drug or experimental procedure within previous 6 months that would interfere with the assessment of ANK-700
• Patients who are pregnant or breastfeeding
Drug: ANK-700, Drug: Placebo
Multiple Sclerosis, Relapsing Remitting Multiple Sclerosis
autoimmune, multiple sclerosis, relapsing remitting ms
UT Southwestern
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Study of CLR 131 in Select B-Cell Malignancies (CLOVER-1) and Pivotal Expansion in Waldenstrom Macroglobulinemia (CLOVER-WaM)

Part A of this study evaluates CLR 131 in patients with select B-cell malignancies (multiple myeloma( MM), indolent chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), lymphoplasmacytic lymphoma (LPL)/Waldenstrom Macroglobulinemia (WM), marginal zone lymphoma (MZL), mantle cell lymphoma (MCL), diffuse large B-cell lymphoma (DLBCL), and central nervous system lymphoma (CNSL) who have been previously treated with standard therapy for their underlying malignancy. Part B (CLOVER-WaM) is a pivotal efficacy study evaluating IV administration of CLR 131 in patients with WM that have received at least two prior lines of therapy.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Larry Anderson
102991
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT02952508
STU-2021-0668
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[CLOVER-1]
Inclusion Criteria:
All Patients
• Histologically or cytologically confirmed MM; Patients with primary or secondary CNSL may be enrolled.
• ECOG performance status of 0 to 2
• 18 years of age or older
• Life expectancy of at least 6 months
• Platelets ≥ 75,000/µL (if full-dose anticoagulation therapy is used, platelets ≥ 100,000/µL are required)
• WBC count ≥ 3000/µL
• Absolute neutrophil count ≥ 1500/µL
• Hemoglobin ≥ 9 g/dL (last transfusion, if any, must be at least 1 week prior to study registration, and no transfusions are allowed between registration and dosing)
• Estimated glomerular filtration rate ≥ 30 mL/min/1.73 m2
• Aspartate aminotransferase (AST) and Alanine aminotransferase (ALT) ≤ 2.5 × upper limit of normal (ULN)
• Bilirubin < 1.5 × ULN
• International normalized ratio (INR) < 2.5
• If patient is on full-dose anticoagulation therapy, the anticoagulation therapy must be reversible and reversal of the anticoagulation therapy must not be life-threatening, as judged by the Investigator
• Patients who have undergone stem cell transplant must be at least 100 days from transplant Patients with Multiple Myeloma
• At least 5 prior regimens, which must include at least 1 approved proteasome inhibitor (bortezomib, carfilzomib, or ixazomib), at least 1 approved immunomodulatory agent (thalidomide, lenalidomide, or pomalidomide), and at least 1 approved monoclonal antibody (e.g., daratumumab or elotuzumab) with or without maintenance therapy, unless patients are intolerable to such agents or ineligible to receive such agents.
• At least triple-class refractory (refractory to a proteasome inhibitor, immunomodulatory agent, and a monoclonal antibody)
• Progressive disease defined by any of the following:
• 25% increase in serum M-protein from the lowest response value during (or after) last therapy and/or absolute increase in serum M-protein of ≥ 0.5 g/dL
• 25% increase in urine M-protein from the lowest response value during (or after) last therapy and/or absolute increase in urine M-protein of ≥ 200 mg/24 h
• 25% increase in bone marrow plasma cell percentage from the lowest response value during (or after) last therapy. Absolute bone marrow plasma cell percentage must be ≥ 10% unless prior CR when absolute bone marrow plasma cell percentage must be ≥ 5%.
• 25% increase in serum FLC level from the lowest response value during (or after) last therapy; the absolute increase must be > 10 mg/dL
• New onset hypercalcemia > 11.5 mg/dL
• Failure to obtain a partial response or better to current treatment, or cannot further improve their response to current treatment
• Appearance of new extramedullary disease
• Measurable disease defined by any of the following:
• Serum M-protein > 0.5 g/dL
• Urine M-protein > 200 mg/24 h
• Serum FLC assay: Involved FLC level ≥ 10 mg/dL provided serum FLC ratio is abnormal. [CLOSED] Patients with Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma, Lymphoplasmacytic Lymphoma/Waldenstom Macroglobulinemia, or Marginal Zone Lymphoma
• Prior treatment with at least 2 prior regimens, which may include chemotherapy, an approved anti-CD20 antibody with or without maintenance therapy, and an approved targeted agent, unless patients are ineligible to receive such agents
• Patients with Helicobacter pylori+ mucosa-associated lymphoid tissue lymphoma must have received 1 prior antibiotic regimen for H pylori
• At least 1 measurable nodal lesion with longest diameter > 15 mm or 1 measurable extranodal lesion (eg, hepatic nodule) with longest diameter > 10 mm. Additional parameters (e.g., measurable IgM for patients with Lymphoplasmacytic Lymphoma) may be allowed if they meet current NCCN guidelines for symptomatic disease. Patients with uptake by FDG-PET scan may be allowed with prior approval of Sponsor. [CLOSED] Patients with Mantle Cell Lymphoma
• Prior treatment with at least 1 prior regimen
• At least 1 measurable nodal lesion with longest diameter > 15 mm or 1 measurable extranodal lesion (eg, hepatic nodule) with longest diameter > 10 mm. Patients with uptake by FDG-PET scan may be allowed with prior approval of Sponsor. [CLOSED] Patients with Diffuse Large B-Cell Lymphoma
• Relapsed or refractory to combination chemotherapy for DLBCL that contains rituximab and an anthracycline; or is intolerable to such agents. Relapsed disease is defined as either recurrence of disease after a CR or PD after achieving a partial response (PR) or SD. Refractory disease is defined as failure to achieve at least SD with any 1 line of therapy or with PD ≤ 3 months of the most recent chemotherapy regimen.
• At least 1 measurable nodal lesion with longest diameter > 15 mm or 1 measurable extranodal lesion (eg, hepatic nodule) with longest diameter > 10 mm. Patients with uptake by FDG-PET scan may be allowed with prior approval of Sponsor. Patients with CNS Lymphoma
• Must have biopsy-proven disease and must have received at least one prior intervention for their disease.
• Must be at least two weeks from CNS biopsy before administration of CLR 131.
• Must have at least one lesion with enhancement on brain imaging.
• Stable (or decreasing) dose of corticosteroids or anti-convulsant medication for at least 7 days prior to dosing [CLOVER-1]
Exclusion Criteria:

• Ongoing Grade 2 or greater toxicities due to previous therapies. Stable, tolerable Grade 2 AEs (eg, neuropathy) may be allowed.
• Prior external-beam RT resulting in greater than 20% of total bone marrow receiving greater than 20 Gy.
• Prior total body or hemi-body irradiation. Patients who have received prior low-dose total body or hemi-body irradiation may be allowed on a case-by-case basis after discussion with Sponsor (considerations may include factors such as time since irradiation, total lifetime accumulated dose, etc.)
• Extradural tumor in contact with the spinal cord or tumor located where swelling in response to therapy may impinge upon the spinal cord
• For patients with CLL/SLL, LPL, or MZL, transformation to a more aggressive form of NHL
• Ongoing chronic immunosuppressive therapy
• Clinically significant bleeding event within prior 6 months
• Ongoing anti-platelet therapy (except low-dose aspirin [eg, 81 mg daily] for cardioprotection)
• Anti-cancer therapy within two weeks of initial CLR 131 infusion. Low dose dexamethasone for symptom management is allowed
• Radiation therapy, chemotherapy, immunotherapy, or investigational therapy within 2 weeks of eligibility-defining bone marrow biopsy.
• For patients with primary or secondary CNSL, active bleeding in the tumor bed and/or uncontrolled seizure activity [CLOVER-WaM] Inclusion Criteria
• Histologically or cytologically confirmed WM. Patients with a diagnosis of LPL may be enrolled with prior Sponsor approval.
• Patient has an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 to 2 (Appendix C)
• Patient is 18 years of age or older
• Life expectancy of at least 6 months
• Received at least two prior lines of therapy for WM
• Measurable IgM (above upper limit of normal) OR at least one measurable nodal lesion with longest diameter > 15 mm or one measurable extranodal lesion (e.g., hepatic nodule) with longest diameter > 10 mm [CLOVER-WaM] Exclusion Criteria
• Ongoing Grade 2 or greater toxicities due to previous therapies, excluding alopecia.
• Prior external-beam RT resulting in greater than 20% of total bone marrow receiving greater than 20 Gy.
• Prior total body or hemi-body irradiation. Patients who have received prior low-dose total body or hemi-body irradiation may be allowed on a case-by-case basis after discussion with Sponsor (considerations may include factors such as time since irradiation, total lifetime accumulated dose, etc.)
• Patients with second malignancies in addition to WM, if the second malignancy has required therapy in the last 2 years or is not in remission; exceptions to this criterion include successfully treated non-metastatic basal cell or squamous cell skin carcinoma, or prostate cancer that does not require therapy
• Anti-cancer therapy within two weeks of initial CLR 131 infusion.
• Need for acute treatment of WM (e.g., those with hyperviscosity)
Drug: CLR 131 single dose, Drug: CLR 131 multiple dose, Drug: CLR 131 fractionated dose
Multiple Myeloma, Waldenström Macroglobulinemia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Marginal Zone Lymphoma, Mantle Cell Lymphoma, Diffuse Large B Cell Lymphoma, Non-Hodgkins Lymphoma, Central Nervous System Lymphoma, Lymphoplasmacytic Lymphoma
Waldenstrom Macroglobulinemia, Non-Hodgkin Lymphoma, NHL, Relapsed, Refractory, Novel class, Pivotal, Phase 3, Hematologic disease, Neoplasm, Plasma cell neoplasms, Paraproteinemias, Lymphoma, Immunoproliferative disorder, Blood protein disorders, Lymphoproliferative disorders, Antineoplastic agents
UT Southwestern
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Follow-up of the PEPITES Study to Evaluate Long-term Efficacy and Safety of Viaskin Peanut in Children (PEOPLE)

This is an open-label, follow-up study for subjects who completed the PEPITES study. Subjects will be offered enrollment in this follow-up study to receive Viaskin Peanut 250 μg for 2 additional years if previously on active treatment in the PEPITES study, or for 3 years if previously on placebo in the PEPITES study.
Call 214-648-5005
studyfinder@utsouthwestern.edu
John Bird
108478
All
Not specified
Phase 3
This study is NOT accepting healthy volunteers
NCT03013517
STU 122016-012
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Inclusion Criteria:

• Subjects who completed the PEPITES study.
Exclusion Criteria:

• Generalized dermatologic disease (for example, active atopic dermatitis, uncontrolled generalized active eczema, ichthyosis vulgaris) extending widely on the skin and especially on the back or arms with no intact zones to apply the Viaskin patches.
• Diagnosis of asthma that evolved to severe, unstable or uncontrolled asthma.
Drug: Viaskin Peanut 250µg
Peanut Allergy, Other
Allergy, food allergy, viaskin, peanut, epicutaneous
Children’s Health
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Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)

This Pediatric MATCH screening and multi-sub-study phase II trial studies how well treatment that is directed by genetic testing works in pediatric patients with solid tumors, non-Hodgkin lymphomas, or histiocytic disorders that have progressed following at least one line of standard systemic therapy and/or for which no standard treatment exists that has been shown to prolong survival. Genetic tests look at the unique genetic material (genes) of patients' tumor cells. Patients with genetic changes or abnormalities (mutations) may benefit more from treatment which targets their tumor's particular genetic mutation, and may help doctors plan better treatment for patients with solid tumors or non-Hodgkin lymphomas.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Avanthi Shah
74989
All
12 Months to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03155620
STU 072017-080
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Inclusion Criteria:

• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients must be >= 12 months and =< 21 years of age at the time of study enrollment
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients with recurrent or refractory solid tumors, including non-Hodgkin lymphomas, histiocytoses (e.g. langerhans cell histiocytosis [LCH], juvenile xanthogranuloma [JXG], histiocytic sarcoma), and central nervous system (CNS) tumors are eligible; patients must have had histologic verification of malignancy at original diagnosis or relapse except in patients with intrinsic brain stem tumors, optic pathway gliomas, or patients with pineal tumors and elevations of cerebrospinal fluid (CSF) or serum tumor markers including alpha-fetoprotein or beta-human chorionic gonadotropin (HCG); in cases where patient enrolls prior to histologic confirmation of recurrent disease, patient is ineligible and should be withdrawn from study if histology fails to confirm recurrence; please note: Patients with Hodgkin lymphoma and plexiform neurofibroma are not eligible
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Tumor Testing Requirement: Tumor sample availability requirement for stage 1 of Pediatric MATCH (patients enrolled from start of study in July 2017 through 12/31/21); Patients must have an formalin-fixed paraffin-embedded (FFPE) tumor sample available for MATCH study testing from a biopsy or surgery that was performed at any point after initial tumor recurrence/progression, or be planned to have a procedure to obtain such a sample that is considered to be of potential benefit by the treating clinicians; a tumor sample from a clinically performed diagnostic (pre-treatment) biopsy will be acceptable for enrollment onto Pediatric MATCH only for children with high-grade gliomas of the brainstem (diffuse intrinsic pontine gliomas) or thalamus
• Please note: Samples that have been decalcified using standardly utilized acid-based decalcification methods are not generally suitable for MATCH study testing; the nucleic acids will have been degraded in the decalcification process
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Tumor molecular profiling report availability requirement for Stage 2 of Pediatric MATCH (patients enrolled starting 2022): In stage 2 of the study, no tumor samples will be submitted for centralized clinical tumor profiling; instead, a tumor molecular profiling report from a College of American Pathologists (CAP)/ Clinical Laboratory Improvements Amendments (CLIA)-approved testing laboratory must be submitted for review by the Molecular Review Committee (MRC)
• This molecular profiling must have been performed on a tumor sample that was obtained at any point after initial tumor recurrence/progression and must be accompanied by a pathology report for the same tumor specimen; a molecular profiling report for a diagnostic (pre-treatment) tumor sample will be acceptable for enrollment onto Pediatric MATCH only for children with high-grade gliomas of the brainstem (diffuse intrinsic pontine gliomas) or thalamus. In the event that molecular profiling reports are available from multiple timepoints, the most recent report should be prioritized for study submission
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age); note: neurologic deficits in patients with central nervous system (CNS) tumors must have been stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients must have radiographically measurable disease; measurable disease based on imaging obtained less than or equal to 56 days prior to enrollment; patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG) positive (+) evaluable disease are eligible; measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard magnetic resonance imaging (MRI) or computed tomography (CT)
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or CSF
• Previously radiated lesions that have not demonstrated clear progression post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: NOTE: patient does not need to meet all subprotocol criteria at time of enrollment onto the APEC1621SC screening protocol, but will need to meet all criteria prior to enrollment on any assigned treatment subprotocol. Patients must be enrolled onto a subprotocol within 2 weeks (14 days) of treatment assignment
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age); Note: neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: At the time of treatment with subprotocol specified therapy, the patients must have radiographically measurable disease; patients with neuroblastoma who do not have measurable disease but have MIBG+ evaluable are eligible; measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard MRI or CT
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or CSF
• Previously radiated lesions that have not demonstrated clear progression post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for RECIST 1.1
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: At the time of enrollment onto a subprotocol, the following general criteria for initiation of therapy will be required:
• Patients must have fully recovered from the acute toxic effects of all prior anticancer therapy and must meet the following minimum duration from prior anticancer directed therapy prior to enrollment to the subprotocol; if after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
• Cytotoxic chemotherapy or other anticancer agents known to be myelosuppressive: for agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment >= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Anticancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil counts [ANC]): >= 7 days after the last dose of agent; for agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. Neulasta) or 7 days for short-acting growth factor; for agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair and the study-assigned research coordinator
• Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
• Stem cell infusions (with or without total-body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, natural killer (NK) cells, dendritic cells, etc.)
• X-ray therapy (XRT)/External Beam Irradiation including Protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation; note: radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-MIBG): >= 42 days after systemically administered radiopharmaceutical therapy
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: For patients with solid tumors without known bone marrow involvement:
• Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
• Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
• Age: 1 to < 2 years; maximum serum creatinine (mg/dL): male 0.6; female 0.6
• Age: 2 to < 6 years; maximum serum creatinine (mg/dL): male 0.8; female 0.8
• Age: 6 to < 10 years; maximum serum creatinine (mg/dL): male 1; female 1
• Age: 10 to < 13 years; maximum serum creatinine (mg/dL): male 1.2; female 1.2
• Age: 13 to < 16 years; maximum serum creatinine (mg/dL): male 1.5; female 1.4
• Age: >= 16 years; maximum serum creatinine (mg/dL): male 1.7; female 1.4
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Serum glutamate pyruvate transaminase (SGPT) (alanine transferase [ALT]) =< 135 U/L (for the purpose of this study, the ULN for SGPT is 45 U/L)
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Patients must be able to swallow intact capsules/tablets, unless otherwise specified in the subprotocol to which they are assigned
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Agent specific limitations on prior therapy will be included with specific treatment subprotocols
Exclusion Criteria:

• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies, or because there is currently no available information regarding human fetal or teratogenic toxicities; pregnancy tests must be obtained in females who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Concomitant medications
• Corticosteroids: at the time of consent and enrollment to regimen specific subprotocols, patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment to the subprotocol will not be eligible; if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Investigational drugs: patients must meet criteria for prior therapy at the time of consent and enrollment to a subprotocol; other investigational agents may not be administered to patients while they are receiving study drug as part of a subprotocol
• Anticancer agents: patients must meet criteria for prior therapy at the time of consent and enrollment to a subprotocol; other investigational agents may not be administered to patients while they are receiving study drug as part of a subprotocol
• Anti-GVHD agents post-transplant: patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Patients who have an uncontrolled infection are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Patients who have had a prior solid organ transplant are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Additional agent specific criteria will be included with specific treatment subprotocols
Procedure: Biopsy, Procedure: Biospecimen Collection, Drug: Ensartinib, Drug: Erdafitinib, Other: Laboratory Biomarker Analysis, Drug: Larotrectinib Sulfate, Procedure: Mutation Carrier Screening, Drug: Olaparib, Drug: Palbociclib, Other: Pharmacological Study, Drug: Samotolisib, Drug: Selpercatinib, Drug: Selumetinib Sulfate, Drug: Tazemetostat, Drug: Tipifarnib, Drug: Ulixertinib, Drug: Vemurafenib
Recurrent Childhood Rhabdomyosarcoma, Recurrent Neuroblastoma, Malignant Glioma, Recurrent Osteosarcoma, Recurrent Malignant Solid Neoplasm, Advanced Malignant Solid Neoplasm, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Wilms Tumor, Recurrent Glioma, Refractory Malignant Solid Neoplasm, Stage III Soft Tissue Sarcoma AJCC v7, Stage IV Soft Tissue Sarcoma AJCC v7, Histiocytic Sarcoma, Juvenile Xanthogranuloma, Recurrent Langerhans Cell Histiocytosis, Recurrent Peripheral Primitive Neuroectodermal Tumor, Refractory Langerhans Cell Histiocytosis, Refractory Neuroblastoma, Rhabdoid Tumor, Stage III Osteosarcoma AJCC v7, Stage IV Osteosarcoma AJCC v7, Stage IVA Osteosarcoma AJCC v7, Stage IVB Osteosarcoma AJCC v7, Refractory Non-Hodgkin Lymphoma, Recurrent Medulloblastoma, Recurrent Non-Hodgkin Lymphoma, Refractory Malignant Germ Cell Tumor, Langerhans Cell Histiocytosis, Recurrent Rhabdoid Tumor, Recurrent Soft Tissue Sarcoma, Refractory Medulloblastoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Recurrent Malignant Germ Cell Tumor, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Non-Hodgkin Lymphoma, Refractory Ewing Sarcoma, Refractory Glioma, Refractory Hepatoblastoma, Refractory Rhabdomyosarcoma, Recurrent Ependymoma, Refractory Primary Central Nervous System Neoplasm, Recurrent Primary Central Nervous System Neoplasm
Children’s Health
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Safety and Efficacy Study of Viaskin Peanut in Peanut-allergic Young Children 1-3 Years of Age (EPITOPE)

The study aims to assess the safety and efficacy of Viaskin Peanut to induce desensitization to peanut in peanut-allergic children 1 to 3 years of age after a 12-month treatment by EPicutaneous ImmunoTherapy (EPIT).
Call 214-648-5005
studyfinder@utsouthwestern.edu
Christopher Parrish
168280
All
1 Year to 3 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03211247
STU 022017-070
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Key
Inclusion Criteria:

• Male or female from 1-3 years of age;
• Physician-diagnosed peanut allergy;
• Peanut-specific IgE level > 0.7 kU/L;
• Positive peanut SPT with a largest wheal diameter ≥ 6 mm;
• Positive DBPCFC at ≤ 300 mg peanut protein; Key
Exclusion Criteria:

• Uncontrolled asthma;
• History of severe anaphylaxis to peanut;
• Prior immunotherapy to any food or other immunotherapy;
• Generalized severe dermatologic disease;
Biological: Viaskin Peanut 250 mcg, Biological: Viaskin Peanut 100 mcg, Biological: Placebo
Peanut Allergy, Other
EPIT, Epicutaneous, Immunotherapy, Viaskin
Children’s Health
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Conditioning SCID Infants Diagnosed Early (CSIDE)

The investigators want to study if lower doses of chemotherapy will help babies with SCID to achieve good immunity with less short and long-term risks of complications after transplantation. This trial identifies babies with types of immune deficiencies that are most likely to succeed with this approach and offers them transplant early in life before they get severe infections or later if their infections are under control. It includes only patients receiving unrelated or mismatched related donor transplants. The study will test if patients receiving transplant using either a low dose busulfan or a medium dose busulfan will have immune recovery of both T and B cells, measured by the ability to respond to immunizations after transplant. The exact regimen depends on the subtype of SCID the patient has. Donors used for transplant must be unrelated or half-matched related (haploidentical) donors, and peripheral blood stem cells must be used. To minimize the chance of graft-versus-host disease (GVHD), the stem cells will have most, but not all, of the T cells removed, using a newer, experimental approach of a well-established technology. Once the stem cell transplant is completed, patients will be followed for 3 years. Approximately 9-18 months after the transplant, vaccinations will be administered, and a blood test measuring whether your child's body has responded to the vaccine will be collected.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Victor Aquino
10208
All
up to 2 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03619551
STU-2018-0210
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Inclusion Criteria:
1. Infants with SCID, either typical or leaky or Omenn syndrome. 1. Typical SCID is defined as either of the following
• Absence or very low number of T cells (CD3+ T cells <300/microliter AND no or very low T cell function (<10% of lower limit of normal) as measured by response to phytohemagglutinin OR
• Presence of maternally derived T cells 2. Leaky SCID is defined as the following • Absence of maternally derived T cells • AND either one or both of the following (i, ii): i) <50% of lower limit of normal T cell function as measured by response to PHA OR <30% of lower limit of normal T cell function as measured by response to CD3 ii) Absent or <10% of lower limit of normal proliferative responses to candida and tetanus toxoid antigens (must document post vaccination or exposure for this criterion to apply) • AND at least two of the following (i through iii): i) CD3 T cells < 1500/microliter ii) >80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (at < 4 years of age) AND/OR are oligoclonal T iii) Low TRECs and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower level of normal. 3. Omenn syndrome • Generalized skin rash
• Maternal lymphocytes tested for and not detected.
• >80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (<2 years of age)
• Absent or low (up to 30% lower limit of normal (LLN)) T cell proliferation to antigens (Candida, tetanus) to which the patient has been exposed IF: Proliferation to antigen was not performed, but at least 4 of the following 8 supportive criteria, at least one of which must be among those marked with an asterisk (*) below are present, the patient is eligible as Omenn Syndrome. 1. Hepatomegaly 2. Splenomegaly 3. Lymphadenopathy 4. Elevated IgE 5. Elevated absolute eosinophil count 6. *Oligoclonal T cells measured by CDR3 length or flow cytometry (upload report) 7. *Proliferation to PHA is reduced to < 50% of lower limit of normal (LLN) or SI < 30 8. *Low TRECs and/or percentage of CD4+/RA+ CD31+ or CD4+/RA+ CD62L+ cells below the lower level of normal 2. Documented mutation in one of the following SCID-related genes a. Cytokine receptor defects (IL2RG, JAK3) b. T cell receptor rearrangement defects (RAG1, RAG2) 3. No available genotypically matched related donor (sibling) 4. Availability of a suitable donor and graft source 1. Haploidentical related mobilized peripheral blood cells 2. 9/10 or 10/10 allele matched (HLA-A, -B, -C, -DRB1, -DQB1) volunteer unrelated donor mobilized peripheral blood cells 5. Age 0 to 2 years at enrollment Note: to ensure appropriate hepatic metabolism, age at time of busulfan start: For IL2RG/JAK3: 8 weeks For RAG1/RAG2: 12 weeks 6. Adequate organ function defined as: 1. Cardiac: Left ventricular ejection fraction (LVEF) at rest ≥ 40% or, shortening fraction (SF) ≥ 26% by echocardiogram. 2. Hepatic: Total bilirubin < 3.0 x the upper limit of normal (ULN) for age (patients who have been diagnosed with Gilbert's Disease are allowed to exceed this limit) and AST and ALT < 5.0 x ULN for age. 3. Renal: GFR estimated by the updated Schwartz formula ≥ 90 mL/min/1.73 m2. If the estimated GFR is < 90 mL/min/1.73 m2, then renal function must be measured by 24-hour creatinine clearance or nuclear GFR, and must be > 50 mL/min/1.73 m2. 4. Pulmonary No need for supplemental oxygen and O2 saturation > 92% on room air at sea level (with lower levels allowed at higher elevations per established center standard of care).
Exclusion Criteria:
1. Presence of any serious life-threatening or opportunistic infection at time of enrollment and prior to the initiation of the preparative regimen. Serious infections as defined below that occur after enrollment must be reported immediately to the Study Coordinating Center, and enrollment will be put on hold until the infection resolves. Ideally enrolled subjects will not have had any infection. If patients have experienced infections, these must have resolved by the following definitions: a. Bacterial i. Positive culture from a sterile site (e.g. blood, CSF, etc.): Repeat culture(s) from same site must be negative and patient has completed appropriate course of antibacterial therapy (typically at least 10 days). ii. Tissue-based clinical infection (e.g. cellulitis): Complete resolution of clinical signs (e.g. erythema, tenderness, etc.) and patient has completed appropriate course of antibacterial therapy (typically at least 10 days). iii. Pneumonia, organism not identified by bronchoalveolar lavage: Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.) and patient has completed appropriate course of antibacterial therapy (typically at least 10 days). If possible, radiographic resolution should also be demonstrated. b. Fungal i. Positive culture from a sterile site (e.g. blood, CSF, etc.): Repeat culture(s) from same site is negative and patient has completed appropriate course of antifungal therapy (typically at least 14 days). The patient may be continued on antifungal prophylaxis following completion of the treatment course. c. Pneumocystis i. Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.) and patient has completed appropriate course of therapy (typically at least 21 days). If possible, radiographic resolution should also be demonstrated. The patient may be continued on prophylaxis following completion of the treatment course. d. Viral i. Viral PCRs from previously documented sites (blood, nasopharynx, CSF) must be re-tested and are negative. ii. If re-sampling a site is not clinically feasible (i.e. BAL fluid): Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.). If possible, radiographic resolution should also be demonstrated. 2. Patients with HIV or HTLV I/II infection will be excluded.
Drug: Busulfan, Device: Cell processing for TCRαβ+/CD19+ depletion
SCID, Other
Children’s Health
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Cladribine Tablets: Collaborative Study to Evaluate Impact On Central Nervous System Biomarkers in Multiple Sclerosis (CLOCK-MS)

The purpose of this study is to better understand the mechanism of action (MoA) of cladribine tablets by exploring the effect on central nervous system (CNS) and blood biomarkers relevant in the relapsing forms of multiple sclerosis (RMS; to include relapsing-remitting MS [RRMS] or active secondary progressive MS).
Call 214-648-5005
studyfinder@utsouthwestern.edu
Olaf Stuve
58631
All
18 Years to 65 Years old
Phase 4
This study is NOT accepting healthy volunteers
NCT03963375
STU-2020-0127
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Inclusion Criteria:
1. Have a relapsing form of multiple sclerosis (RMS; to include RRMS or active secondary progressive MS) 2. Have had an inadequate response to, or are unable to tolerate, an alternate drug indicated for the treatment of MS 3. Are willing and able to receive at least 2 lumbar punctures 4. Capable of giving signed informed consent
Exclusion Criteria:
1. Have any contraindication for lumbar puncture or any other comorbid conditions that preclude participation 2. Are infected with human immunodeficiency virus (HIV) 3. Have active chronic infections (e.g. hepatitis or tuberculosis) 4. Have been previously treated with cladribine 5. Have previously been treated with ocrelizumab, alemtuzumab, rituximab, or daclizumab 6. Have received treatment with natalizumab during the last 6 months 7. Are currently receiving immunosuppressive or myelosuppressive therapy, e.g., methotrexate, cyclophosphamide, cyclosporine or azathioprine, or chronic treatment with systemic corticosteroids 8. Have moderate or severe hepatic or renal impairment 9. Are pregnant or unwilling or unable to use effective contraception during cladribine tablets dosing and for 6 months after the last dose in each treatment course 10. Are intending to breastfeed on a cladribine tablet treatment day and/or during the 10 days after the last cladribine tablet dose.
Drug: Cladribine
Multiple Sclerosis, Multiple Sclerosis, Relapsing-Remitting
UT Southwestern
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Efficacy and Safety Study of MYOBLOC® in the Treatment of Adult Upper Limb Spasticity

Phase 2/3, randomized, double-blind, placebo-controlled, single-treatment, multicenter trial assessing the efficacy and safety of MYOBLOC for the treatment of upper limb spasticity in adults followed by an open-label extension safety trial.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Fatma Gul
12837
All
18 Years to 80 Years old
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT04815967
STU-2021-0158
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INCLUSION CRITERIA: 1. Able to understand the potential risks and benefits, the study requirements, and provide written informed consent before enrollment into the study; or if unable, the subject's Legally Authorized Representative (LAR) may provide written informed consent. 2. Male or female ≥18 to maximum of 80 years of age, inclusive. 3. Upper limb spasticity due to stroke, or traumatic brain injury, or spinal cord injury that occurred ≥ 6 months prior to randomization. Eligible subjects may have upper limb monoplegia or hemiplegia. Subjects with cerebral palsy are eligible for study enrollment. 4. Modified Ashworth Scale (MAS) scores of ≥2 in at least two muscle groups inclusive of the elbow, wrist, and finger flexors at screening and baseline. 5. In the Investigator's opinion, the subject will be available and able to comply with the study requirements for at least 1 year, based on the subject's overall health and disease prognosis. 6. In the Investigator's opinion, the subject will be willing and able to comply with all requirements of the protocol, including completion of study questionnaires. A caregiver may be designated to assist with the physical completion of questionnaires/scales. EXCLUSION CRITERIA: 1. Quadriplegia/tetraplegia, or triplegia with both upper limbs affected. 2. Uncontrolled epilepsy or any type of seizure disorder with a seizure(s) within the previous year. 3. Neuromuscular disorders including, but not limited to, amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), multiple sclerosis (MS), myasthenia gravis, or muscular dystrophy. 4. History of major joint contracture(s), in which, based on the Investigator's assessment, the contracture(s) significantly contributes to joint immobility in the affected upper limb. 5. Unresolved fracture(s) in the affected upper limb. 6. Severe atrophy in the affected upper limb. 7. Known hypersensitivity to botulinum toxins type A or B or to any MYOBLOC solution components. 8. Concomitant use or exposure within 5 half-lives of randomization of the following: aminoglycoside antibiotics, curare-like agents, or other agents that may interfere with neuromuscular function. 9. Treatment with a neurolytic agent (e.g., phenol, alcohol blocks) to the affected upper limb within 1 year before randomization. 10. Presence of a spinal stimulator or intrathecal baclofen pump that has not been turned off within 30 days prior to screening. 11. Changes to treatment regimen or any new treatment with oral antispasmodics and/or muscle relaxants within 30 days prior to randomization. 12. Initiation of physical and/or occupational therapy <30 days before randomization. Subjects receiving physical and/or occupational therapy ≥30 days before randomization must be willing to maintain their therapy regimen through Week 4 of the Double-Blind Period. 13. Prior botulinum toxin type A (BoNT/A) or B (BoNT/B) treatment in the affected upper limb within 24 weeks before screening. Prior BoNT/A or BoNT/B treatment in areas other than the affected upper limb is not exclusionary but must have occurred at least 12 weeks before screening. Prior toxin exposure must have been well tolerated and without any significant long-term side effects in the case of repeated prior exposure. 14. Subjects should not receive nor have any plans to receive any botulinum toxin treatment, other than the study drug (MYOBLOC), from the time that informed consent is obtained until participation in the study is complete. 15. Severe dysphagia (i.e., inability to swallow liquids, solids or both without choking or medical intervention), or dysphagia with a history of aspiration pneumonia, within 6 months before screening. 16. Prior surgery to treat spasticity in the affected upper limb (i.e., tendon lengthening or tendon transfer). 17. Any anticipated or scheduled surgery during the study period, with the exception of dermatological procedures performed under local anesthesia for the purposes of removing precancerous and cancerous lesions. 18. Major surgery within 30 days before screening. 19. Pregnancy or breastfeeding. 20. Females of childbearing potential must agree to practice a medically acceptable method of contraception (e.g., intrauterine device, hormonal contraception started at least one full cycle before study enrollment, or barrier method in conjunction with spermicide) for the duration of the study (including 2 months after study completion). For the purposes of this study, all females are considered to be of childbearing potential unless they are confirmed by the Investigator to be post-menopausal (at least 1 year since last menses and laboratory test confirmation), biologically sterile, or surgically sterile (e.g., hysterectomy with bilateral oophorectomy, tubal ligation). 21. History of drug or alcohol abuse within 6 months before screening. 22. Obstructive pulmonary disease with forced expiratory volume in 1 second (FEV1)/forced vital capacity (FVC) <70%. 23. Slow vital capacity (SVC) <60% of predicted. 24. Chronic or current use of inhaled corticosteroids. 25. Ventilator dependence (i.e., 24-hour ventilator dependence when intubated, or due to a failure to wean the subject from the ventilator while hospitalized in the intensive care unit or respiratory care center). Subjects who use oxygen on an as-needed basis or during sleeping hours only via a nasal cannula are eligible for the study. 26. Infection at the planned sites of injection. 27. Treatment with an investigational drug, device, or biological agent within 30 days before screening or while participating in this study. 28. Malignancy diagnosed 3 months before screening. 29. Has one or more screening clinical laboratory test values outside the reference range that, in the opinion of the Investigator, are clinically significant, or any of the following :
• Serum creatinine >1.5 times the upper limit of normal (ULN);
• Serum total bilirubin > 1.5 times ULN;
• Serum alanine aminotransferase or aspartate aminotransferase >2 times ULN. 30. Has any of the following cardiac findings at screening:
• Abnormal ECG that is, in the Investigator's opinion/evaluation, clinically significant;
• PR interval >220 ms;
• QRS interval >130 ms;
• QTcF interval >450 ms (for men), or >470 ms (for women) (QT corrected using Fridericia's method);
• Second-or third-degree atrioventricular block;
• Any rhythm, other than sinus rhythm, that is interpreted or assessed by the Investigator to be clinically significant. 31. Any other medical illness, condition, or clinical finding that, in the opinion of the Investigator and/or the Sponsor, would put the subject at undue risk.
Drug: Phase 2, Low Dose MYOBLOC, Drug: Phase 2, High Dose MYOBLOC, Drug: Phase 2, Placebo, Drug: Phase 3, MYOBLOC, Drug: Phase 3, Placebo
Multiple Sclerosis, Cerebral Palsy, Traumatic Brain Injury, Cerebrovascular Accident, Brain and Nervous System, Bones and Joints, Spasticity, Cervical Spinal Cord Injury
Stroke, Traumatic Brain Injury, Monoplegia, Hemiplegia, Upper limb, Multiple Sclerosis, Cerebral Palsy, Cervical Spinal Cord Injury
UT Southwestern
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Safety and Effectiveness of Nabiximols Oromucosal Spray as Add-on Therapy in Participants With Spasticity Due to Multiple Sclerosis (RELEASE MSS3)

This trial is being conducted to demonstrate the efficacy of nabiximols, compared with placebo, when added to standard of care, in the treatment of muscle spasms associated with multiple sclerosis (MS).
Call 214-648-5005
studyfinder@utsouthwestern.edu
Lindsay Horton
103589
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04203498
STU-2020-0646
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Inclusion Criteria:
Criteria at screening: 1. Participant is male or female aged 18 years or above. 2. Participant has had a diagnosis with any disease subtype of multiple sclerosis (MS), by revised 2017 McDonald criteria, for at least 12 months prior to screening and is expected to remain stable for the duration of the trial. 3. Participant has had treatment with at least 1 optimized oral antispasticity therapy prior to Visit 1 that must include either oral baclofen or oral tizanidine (monotherapy or combination therapy). 4. Participant is currently receiving optimized treatment with at least 1 oral antispasticity medication (baclofen, tizanidine, and/or dantrolene) and has been stable for at least 30 days prior to screening. 5. If the participant is currently receiving an approved MS disease-modifying therapy, it must be at a stable dose for at least 3 months prior to screening and is expected to remain stable for the duration of the trial.
Exclusion Criteria:
1. Participant has any concomitant disease or disorder that has spasticity-like symptoms or that may influence the participant's level of spasticity. 2. Participant has had a relapse of MS within the 60 days prior to screening (Visit 1). 3. Participant is currently using or has used cannabis or a cannabinoid-derived product for medicinal or recreational use (within 30 days of screening) and is unwilling to abstain for the duration of the trial. 4. Participant is currently using botulinum toxin injection for the relief of spasticity (within 6 months of screening) and is unwilling to abstain for the duration of the trial. 5. Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP. 6. Participant is male and fertile unless willing to ensure that he uses male contraception or remains sexually abstinent during the trial and for 3 months thereafter. 7. Participant is female and of childbearing potential unless willing to ensure that she uses a highly effective method of birth control during the trial and for 3 months thereafter. 8. Participant is female and pregnant, lactating, or planning pregnancy during the course of the trial or within 3 months thereafter. 9. Participant has received an IMP within the 30 days prior to screening. 10. Participant has a history of severe psychiatric disorder that may be exacerbated by the use of a cannabinoid-containing product. 11. Participant has any known or suspected history of alcohol or substance abuse (including opiate abuse) or dependence within 1 year prior to screening. 12. Participant is currently taking drugs that are solely metabolized by UGT1A9 and UGT2B7 13. Participant is currently taking strong currently taking strong CYP3A4 inducers (e.g., rifampicin, carbamazepine, phenytoin, phenobarbital, St John's Wort)
Drug: Nabiximols, Drug: Placebo
Multiple Sclerosis (MS), Brain and Nervous System
Spasticity
UT Southwestern
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Cutaneous Lupus Registry

Approximately 1.4 million individuals in the United States have systemic lupus erythematosus, and about 85% of these individuals develop skin lesions at some point of their disease. Cutaneous lupus erythematosus represents the skin manifestations of systemic lupus erythematosus, and can appear in people with or without systemic lupus. It is a mentally, physically, and emotionally debilitating disease that affects both the quality of life and social well-being of those affected. The cause of cutaneous lupus is not completely understood, but likely includes multiple factors from our genes and the environment. Multiple genetic studies with small numbers of cutaneous lupus patients have been performed to determine which genes are associated with cutaneous lupus. This study aims to accumulate even larger numbers of patients to confidently identify genes and the proteins they encode that could contribute greatly to the formation of cutaneous lupus. The discovery of these genes and proteins would help not only uncover how cutaneous lupus forms, but also improve our abilities to diagnose this disease and predict its course, and stimulate new drug development.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Benjamin Chong
99998
All
18 Years and over
N/A
This study is also accepting healthy volunteers
NCT01266915
STU 082010-241
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Inclusion Criteria:

• Diagnosed with cutaneous lupus erythematosus and/or systemic lupus erythematosus by clinical, laboratory, and histopathological findings
• Ability to speak and read English or Spanish at a 6th grade reading level (a translator will be available with additional consent forms in Spanish)
• Ability to give written informed consent
Exclusion Criteria:

• Less than 18 years of age, since the characteristics of the disease in these subjects could be very different
• Due to a medication, in which its discontinuation results in the resolution of cutaneous lupus, since the characteristics of the disease in these subjects could be very different
• Medical conditions who do not warrant a skin biopsy
• Unable to give written, informed consent or undergo a skin biopsy and/or venipuncture for any other reason
Lupus Erythematosus, Other Skin
SCLE, CLE, DLE
UT Southwestern
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Patients Treated for SCID (1968-Present)

Individuals with a past diagnosis of severe combined immune deficiency (including many cases of "leaky SCID", Omenn syndrome, and reticular dysgenesis) who have undergone blood and marrow transplant, gene therapy, or enzyme replacement in the past may be eligible for this study. The purpose of study is to look backwards at what has already been done in the. Over 800 patients with SCID are expected to be enrolled, making this one of the largest studies ever to describe outcomes for patients with SCID treated at many different hospitals around North America.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Victor Aquino
10208
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT01346150
STU 032011-168
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Inclusion Criteria:
Strata A, B, and C (Part 1
•Retrospective Study)-
• Individuals with Severe Combined Immune Deficiency (SCID) diagnosis who: --were treated at a location participating in this consortium from 1968 until present, and --are not enrolled in RDCRN PIDTC-6901 (ClinicalTrials.gov ID: NCT01186913).
• Subjects who received HCT/GT/ERT prior to the present date are eligible for the retrospective study. The enrollment criteria for subjects who died prior to definitive therapy are the same as for Strata A, B and C. Stratum A, Typical SCID:
• Individuals who meet the following inclusion criteria and who received HCT are eligible for enrollment into Stratum A of the study:
• Absence or very low number of T cells (CD3 T cells < 300/microliter), and no or very low T cell function (< 10% of lower limit of normal) as measured by response to phytohemagglutinin (PHA) or cells of maternal origin present.
• If maternal cells are present but the patient does not meet criteria for very low T cell function as defined, the assigned reviewers for the potential subject, and if necessary, the full PID-SCID RP will review the laboratory report to determine if criteria of maternal engraftment are met for Protocol 6902.
• Laboratory report of testing for maternal engraftment is required, for evaluation by the PID-SCID RP. Stratum B, Leaky SCID, Omenn Syndrome, Reticular Dysgenesis: Individuals who meet the following criteria are eligible for enrollment into Stratum B of the study: Leaky SCID-
• Maternal lymphocytes tested for and not detected and,
• Either one or both of the following (a,b): a) < 50% of lower limit of normal T cell function (as measured by response to PHA OR < 50% of lower limit of normal T cell function as measured by response to CD3/CD28 antibody, b) Absent or < 30% lower limit of normal proliferative responses to candida and tetanus toxoid antigens postvaccination or exposure,
• AND at least one of the following (a through e): 1. Reduced number of CD3 T cells, 2. > 80% of CD3+ or CD4 T cells are CD45RO+,
• AND/OR >80% of CD3+ or CD4+ T cells are,CD62L negative,
• AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (at < 4 years of age),
• AND/OR are oligoclonal T cells. c) Hypomorphic mutation in IL2RG in a male, or homozygous hypomorphic mutation or compound heterozygosity with at least one hypomorphic mutation in an autosomal SCID-causing gene. d) Low TRECs and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower limit of normal. e) Functional testing in vitro supporting impaired, but not absent, activity of the mutant protein,
• AND does not meet criteria for Omenn Syndrome,
• AND does not have known selective loss of lymphocytes, Ataxia- Telangiectasia, or congenital heart defect associated with lymphopenia, unless a SCID genotype is also present. Omenn Syndrome (OS):
• Generalized skin rash,
• Maternal engraftment tested for and not detected,
• Absent or low (up to 30% of normal) T cell proliferation to antigens to which the patient has been exposed.
• If the proliferation to antigen was not performed, but at least 4 of the following 10 supportive criteria, at least one of which must be among those marked with an asterisk (*) are present, the patient is eligible: hepatomegaly; splenomegaly; lymphadenopathy; elevated IgE; elevated absolute eosinophil count; *oligoclonal T cells measured by CDR3 length or flow cytometry >80% of CD4+ T cells are CD45RO+ ;*proliferation to PHA is reduced <50% of lower limit of normal or SI <30; *proliferative response in mixed leukocyte reaction is reduced to increment cpm < 20% or SI <20; hypomorphic mutation to SCID causing gene; low TRECs and/or percentage of CD 4+/ RA+/CD31+; or CD4+/RA+/CD62L+ cells below the lower limit of normal. Reticular Dysgenesis (RD):
• Absence or very low number of T cells (CD3 T cells <300/microliter),
• No or very low (<10% of lower limit of normal) T cell function (as measured by response to phytohemagglutinin (PHA),
• Severe congenital neutropenia (absolute neutrophil count <200/microliter),
• AND at least one of the following:
• Sensorineural deafness and/or absence of granulopoiesis at bone marrow examination and/or a deleterious AK2 mutation,
• absence of granulopoiesis on bone marrow examination; a pathogenic mutation in the adenylate kinase 2 (AK2) gene identified. Stratum C, SCID with Non-HCT Treatments: -Individuals who meet the following criteria and were treated with PEG-ADA or gene therapy with autologous modified cells are eligible for enrollment into Stratum C (SCID with non-HCT treatments) of the study-
•Any SCID patient previously treated with a thymus transplant (includes intention to treat with HCT, as well as PEG-ADA ERT or gene therapy). Strata A, B, and C (Part 2
•Cross-Sectional Study): Patient inclusion criteria for the cross sectional study: Eligibility for Strata A, B and C are the same as for the retrospective study except that all the patients in the cross-sectional study are currently surviving and are at least 2 years post the most recent class of therapy.
Exclusion Criteria:
Parts 1 and 2
•Retrospective and Cross-Sectional Studies -
• Lack of appropriate testing to rule out HIV infection after 1997 (p24 antigen or more sensitive) or other cause of secondary immunodeficiency,
• Presence of DiGeorge syndrome,
• Most patients with other PIDs such as nucleoside phosphorylase deficiency, ZAP70 deficiency, CD40 ligand deficiency, NEMO deficiency, XLP, cartilage hair hypoplasia or ataxia telangiectasia will not meet the inclusion criteria for Stratum A, B, or C above; however, a patient with one of the above may meet the inclusion criteria for Stratum B and if so will be included-
• MHC Class I and MHC Class II antigen deficiency are excluded,
• Metabolic conditions that imitate SCID or related disorders such as folate transporter deficiency, severe zinc deficiency, transcobalamin deficiency.
SCID, ADA-SCID, XSCID, Leaky SCID, Omenn Syndrome, Reticular Dysgenesis, Unknown Sites
Children’s Health
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Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995

Chronic granulomatous disease (CGD) is an inherited immune system abnormality in which bone marrow transplantation (BMT) has been shown to be curative. However the risks of transplantation are high and not all patients with CGD may need to undergo this high risk procedure. This study will determine the long term medical condition and daily functioning of participants with CGD after a transplant and if possible, compare these results to participants who do not undergo a transplant.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Victor Aquino
10208
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT02082353
STU 012014-053
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Inclusion Criteria:

• Participant Inclusion Criteria (Part 1
•Longitudinal Analysis)
• CGD Patients Undergoing Transplant 1995 to Present with Birth Year In or After 1988 1. CGD Patients will be Defined by both Defective Neutrophil NADPH Oxidase Function and by Clinical History Consistent with CGD Patients must have both of: A functional assay demonstrating abnormal NADPH oxidase function (see A below); AND Clinical history consistent with CGD (see B below). ************************************************************************* Patients must have both "A" and "B": A. Function: Assays of NADPH Oxidase Function I. Dihydrorhodamine (DHR) Assay:
• Blood sample was obtained at a time when patient was clinically stable and not critically ill, with control samples performed simultaneously indicating a qualified assay; and
• Assay unequivocally demonstrates CGD with an stimulation index (SI) SI < 35 or equivalent. Assay report, including mean fluorescence intensity (MFI) from unstimulated and stimulated samples and gating strategy, must be de-identified and provided. OR II. Nitroblue Tetrazolium Oxidation Test (NBT): o Diagnostic of CGD (reported as reduced granulocyte oxidative response). Report must be de-identified and provided. AND B. Clinical History: One or More of the Following:
• Severe and/or recurrent infection (liver, perirectal or lung abscess; pneumonia; adenitis; or osteomyelitis) due to, for example, Staphylococcus aureus, Burkholderia sp, Serratia marcescens, non-albicans Candida sp, Aspergillus sp or other mold; or Nocardia sp or other deep tissue infection characteristic of CGD
• Sterile granulomatous disease in respiratory, gastrointestinal or urogenital tracts; or Crohn's disease-like colitis
• A family history consistent with either X-linked or autosomal recessive CGD In cases where either functional assay (A) or history (B) is equivocal, one or more of the following may be used to confirm a diagnosis of CGD: C. Absent or significantly reduced in expression or abnormal size of any of the 5 phox components (gp91 phox, p47 phox, p22 phox, p67phox, and p40phox) of NADPH oxidase, by either:
• Western blot
• Northern blot OR D. Mutation in a gene encoding one of the 5 phox components (gp91 phox, p47 phox, p22 phox, p67 phox, and p40 phox) of NADPH oxidase that is predictive of a decreased or absent oxidative burst. (Nonsense, frameshift, or previously described missense mutation associated with CGD). Molecular Diagnosis is Desirable In addition, molecular diagnosis (gene sequencing and expression analysis) of CGD is desirable and should be performed when possible. 2. Further Characterization of Oxidase Level, Longitudinal Study, Prospective Cohort Patients who are to undergo transplantation during the study period must be further characterized as oxidase-null or oxidase positive by level of oxidase production by either:
• DHR assay stimulation Index: where SI ≤ 2.5 will be classified as oxidase-null CGD. Those with SI > 2.5 will be classified as oxidase positive CGD. A single validated test that is accepted by the PID-CGD Review Panel is adequate, but testing on two occasions for validation is desirable. OR
• Ferricytochrome C reduction assay of granulocytes with O2 < 2.3 nmoles /106 cells/h classified as oxidase-null CGD. A single validated test that is accepted by the PID-CGD Review Panel is adequate, but testing on two occasions for validation is desirable. OR o Genetic sequencing reporting a mutation that is unequivocally associated to absent oxidase production. (e.g. null mutations) will be classified as oxidase-null CGD (See discussion in Appendix I for how family history, genotype and CGD mutation information will be applied to assigning patients lacking any quantitative oxidase activity measurements to residual oxidase-null or residual oxidase-positive groups). 3. Longitudinal Study, Retrospective Cohort Patients who have already been transplanted will be included regardless of whether further characterization by oxidase level (or genotype/mutation data) is possible or not.
• Non-Transplanted CGD Patients with Birth Year In or After 1988 A non-transplant (conventional therapy) group of CGD subjects will be enrolled in the longitudinal study. The non-transplant subjects will be selected from the potentially eligible (retrospective) patient cohort with diagnosis of CGD treated with conventional non-transplant therapy. Participating sites will enter their entire retrospective cohort of CGD patients having birth year in or after 1988 into the registration cohort for this protocol. Baseline for both non-transplant subjects and HCT subjects for the purpose of comparing survival will be the year of birth. However, for non-transplant subjects, many of the detailed analyses such as infection and autoimmune complication rates will be assessed in the year preceding the date of last contact.
• Participant Inclusion Criteria (Part 2
•Cross-Sectional Analysis) To participate in the Cross-Sectional Analysis, patients must have previously been enrolled into the Longitudinal Analysis of Protocol 6903. All transplanted subjects in the Cross-Sectional Analysis are surviving and shall have at least 3 years of follow-up post-transplant to be included. Non-transplanted CGD subjects will become eligible for consideration for the Cross-Sectional Analysis if they were eligible and enrolled in the retrospective cohort of the Longitudinal Analysis, and if/when they are > 3 years post-diagnosis of CGD. Provision of written informed consent will be required for inclusion in the Cross-Sectional Analysis.
Exclusion Criteria:

• Participant Exclusion Criteria (Longitudinal and Cross- Sectional Analyses)
• Presence of other primary immunodeficiency syndromes that do not meet the clinical and laboratory criteria for CGD.
• Rac2 Deficiency
• Myeloperoxidase Deficiency (MPO Deficiency)
• Glutathione deficiency
• Leukocyte adhesion deficiency syndrome
• Non-transplant subjects:
• The above exclusions pertain.
• In addition, non-transplant subjects will be excluded if the only assessment of oxidase function available is the nitroblue tetrazolium (NBT) test (a non-quantitative test).
Granulomatous Disease, Chronic
Granulomatous Disease, Chronic, Hematopoietic Stem Cell Transplantation (HSCT), bone marrow transplant (BMT), non-transplant, factors associated with best outcomes of transplant in CGD
Children’s Health
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PEDIATRIC SONICS: Pediatric Study of Neuropsychology and Imaging in CNS Demyelinating Syndromes. (SONICS)

Central Nervous System (CNS) demyelinating conditions include multiple sclerosis (MS), Acute Disseminated Encephalomyelitis (ADEM), Neuromyelitis Optica Spectrum Disorder (NMOSD) and Transverse Myelitis (TM). The symptoms of these conditions are quite variable from patient to patient, but can include motor, sensory, visual, gait and cognitive changes. Conventional MRI can be used to look for new anatomic changes, but fails to measure underlying biochemical changes in brain tissue. The purposes of this study are to identify the biologic and anatomic correlations between cognitive profiles and disease activity using MRI imaging techniques.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Benjamin Greenberg
105091
All
12 Years to 18 Years old
This study is also accepting healthy volunteers
NCT03942952
STU-2019-0491
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Inclusion Criteria:
1. Diagnosis of Multiple Sclerosis , ADEM, anti-MOG antibody associated CNS demyelination 2. Age 12 to 18 inclusive at time of enrollment 3. Ability of parent or legal guardian to provide informed consent if participant is under 18. 4. Ability of patients age 12 and older to give assent 5. Completion of the signed HIPPA authorization form by a parent or legal guardian or by participants (18 years of age).
Exclusion Criteria:
1. Known history of traumatic brain injury that required medical care 2. Non-English speaking (based on standardized neuropsychological testing and questionnaires) 3. Claustrophobic, pregnant, the presence of metallic braces, implants or medical devices that are unsafe at 3T or 7T and/or interfere with the MRI/MRS signals
Neuromyelitis Optica, Transverse Myelitis, Acute Disseminated Encephalomyelitis, Multiple Sclerosis, Relapsing-Remitting, Head and Neck
Multiple Sclerosis, NMO, anti-MOG, Acute Disseminated Encephalolmyelitis
UT Southwestern; Children’s Health
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Effect of Pyridostigmine (Mestinon) on Muscle Strength in Myasthenia Gravis

A randomized, placebo-controlled, double-blinded cross-over study evaluating and quantifying the effect of pyridostigmine on muscle strength and symptoms in Myasthenia Gravis (MG)
Call 214-648-5005
studyfinder@utsouthwestern.edu
Steven Vernino
67844
All
18 Years to 90 Years old
Phase 4
This study is NOT accepting healthy volunteers
NCT03510546
STU-2020-0188
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Inclusion Criteria:

• MG verified by a) anti-body, or b) single-fiber EMG and/or decrement on ENG.
Exclusion Criteria:

• Anti-MuSK
• Known cardio-pulmonary disease
• Known neuropathy
• Known myopathy
• Known malignant disease
• Pregnancy or breastfeeding
• Mechanic ileus, urinary tract obstruction, peritonitis De-novo MG Eligibility Criteria
• MG diagnosis < 2 months, no prior antimyasthenic medications Chronic MG Eligibility Criteria
• MG diagnosis > 1 year, and stable pyridostigmine dosis
Drug: Pyridostigmine, Drug: Placebo oral capsule
Myasthenia Gravis
UT Southwestern
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Natural History Study of SCID Disorders

This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions. In order to determine the patient, recipient and transplant-related variables that are most important in determining outcome, study investigators will uniformly collect pre-, post- and peri-transplant (or other treatment) information on all children enrolled into this study. Children will be divided into three strata: - Stratum A: Typical SCID with virtual absence of autologous T cells and poor T cell function - Stratum B: Atypical SCID (leaky SCID, Omenn syndrome and reticular dysgenesis with limited T cell diversity or number and reduced function), and - Stratum C: ADA deficient SCID and XSCID patients receiving alternative therapy including PEG-ADA ERT or gene therapy. Each Group/Cohort Stratum will be analyzed separately.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Victor Aquino
10208
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT01186913
STU 102010-169
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Inclusion Criteria:
Stratum A: Typical SCID (formerly referred to as Classic SCID)- -Subjects who meet the following inclusion criteria and the intention is to treat with allogeneic hematopoietic cell transplant (HCT) are eligible for enrollment into Stratum A (Typical SCID) of the study:
• Absence or very low number of T cells (CD3 T cells <300/microliter) AND
• No or very low T cell function (<10% of lower limit of normal) as measured by response to phytohemagglutinin (PHA) OR
• T cells of maternal origin present. Stratum B: Leaky SCID, Omenn Syndrome, Reticular Dysgenesis- -Subjects who meet the following criteria and the intention is to treat with HCT are eligible for enrollment into Stratum B: Leaky SCID:
• Maternal lymphocytes tested for and not detected AND
• Either one or both of the following (a,b) :
• a.) <50% of lower limit of normal T cell function as measured by response to PHA, OR response to anti-CD3/CD28 antibody
• b.) Absent or <30% of lower limit of normal proliferative responses to candida and tetanus toxoid antigens
• AND at least two of the following (a through e):
• a.) Reduced number of CD3 T cells
• age ≤2 years: <1500/microliter
• age >2 years and ≤4 years: <800/microliter
• age >4 years: <600/microliter
• b.) ≥80% of CD3+ or CD4+ T cells that are CD45RO+
• AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative
• AND/OR >50% of CD3+ or CD4+T cells express HLA-DR (at <4 years of age)
• AND/OR are oligoclonal T cells
• c.) Hypomorphic mutation in IL2RG in a male, or homozygous hypomorphic mutation or compound heterozygosity with ≥1 hypomorphic mutation in an autosomal SCID-causing gene
• d.) Low T Cell Receptor Excision Circles (TRECs) and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower limit of normal.
• e.) Functional testing in vitro supporting impaired, but not absent, activity of the mutant protein, AND
• Does not meet criteria for Omenn Syndrome. Omenn Syndrome:
• Generalized skin rash
• Maternal lymphocytes tested for and not detected; --Note: If maternal engraftment was not assessed and ruled out, the subject is not eligible as Omenn Syndrome.
• ≥80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR
• 80% of CD3+ or CD4+T cells are CD62L negative AND/OR
• 50% of CD3+ or CD4+ T cells express HLA-DR (at <2 years of age);
• Absent or low (< 30% lower limit of normal) T cell proliferation response to antigens (Candida, tetanus) to which the subject has been exposed NOTE: If proliferation to antigen was not performed, but at least 4 of the following 9 supportive criteria, at least one of which must be among those marked with an asterisk (*) below are present, the subject is eligible as Omenn Syndrome:
• Hepatomegaly
• Splenomegaly
• Lymphadenopathy
• Elevated IgE
• Elevated absolute eosinophil count
• *Oligoclonal T cells measured by CDR3 length or flow cytometry
• *Proliferation to PHA is reduced <50% of lower limit of normal or SI <30
• *Hypomorphic mutation in a SCID causing gene
• Low TRECS and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower limit of normal. Reticular Dysgenesis:
• Absence or very low number of T cells (CD3 <300/µL
• No or very low (<10% lower limit of normal) T cell response to PHA
• Severe neutropenia (absolute neutrophil count < 200 /µL) AND
• ≥2 of the following (a,b,c):
• a.) Sensori-neural deafness
• b.) Deficiency of marrow granulopoiesis on bone marrow examination
• c.) A pathogenic mutation in the adenylate kinase 2 (AK2) gene identified. Stratum C: Subjects who meet the following criteria and the intention is to treat with therapy other than allogeneic HCT, primarily PEG-ADA ERT or gene therapy with autologous modified (gene transduced) cells, are eligible for enrollment into Stratum C:
• ADA Deficient SCID with intention to treat with PEG-ADA ERT
• ADA Deficient SCID with intention to treat with gene therapy
• X-linked SCID with intention to treat with gene therapy
• Any SCID patient previously treated with a thymus transplant (includes intention to treat with HCT, as well as PEG-ADA ERT or gene therapy)
• Any SCID patient who received therapy for SCID deemed "non-standard" or "investigational", including in utero procedures.
Exclusion Criteria:
-Subjects who meet any of the following exclusion criteria are disqualified from enrollment in Strata A, B, or C of the study:
• Presence of an Human Immunodeficiency Virus (HIV) infection (by PCR) or other cause of secondary immunodeficiency
• Presence of DiGeorge syndrome
• MHC Class I and MHC Class II antigen deficiency, and
• Metabolic conditions that imitate SCID or related disorders such as folate transporter deficiency, severe zinc deficiency or transcobalamin deficiency.
XSCID, Leaky SCID, Omenn Syndrome, Reticular Dysgenesis, Severe Combined Immunodeficiency (SCID), ADA SCID, Unknown Sites
Severe Combined Immunodeficiency (SCID), natural history study, SCID treatment
Children’s Health
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Morphea in Adults and Children (MAC) Cohort Study: A Morphea Registry and DNA Repository (MAC)

The Morphea in Adults and Children (MAC) cohort is the first registry for both children and adults with morphea (also known as localized scleroderma) in the country. The purpose of the registry is to learn more about morphea, specifically: - How morphea behaves over time - How frequently specific problems occur along with morphea (for example, arthritis) - Whether morphea has an autoimmune background
Call 214-648-5005
studyfinder@utsouthwestern.edu
Heidi Jacobe
54629
All
up to 90 Years old
N/A
This study is also accepting healthy volunteers
NCT01808937
STU 112010-028
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Inclusion Criteria:
1. Patient must have a clinical diagnosis of morphea confirmed by the primary investigator and by histopathological examination. 2. Ages 0-90 years old 3. Children must weigh more than 20 lbs. in order to satisfy Children's Medical Center policy for the maximum amount of blood drawn in a 24 hour period. 4. Patient or legal guardian must be able to speak and read at a 6th grade reading level. 5. Both male and female patients will be eligible 6. All races and ethnic backgrounds will be included 7. Relationships to proband: All patients with morphea will be included. A patient's family history will be reviewed and if there is a family history of morphea or systemic sclerosis then we will give the study patient the investigator's contact information and ask the family member to call the study team to answer any questions and enroll them in the study if they choose to do so. 8. Ability to give informed consent: Patients must be able to give informed consent or they will give assent with parent or guardian consent as a minor to be a part of the morphea registry.
Exclusion Criteria:

•Patients who have been coded as morphea (701.0), but do not have morphea/localized scleroderma (examples: steroid atrophy, acquired keratoderma, keloids, nephrogenic fibrosing dermopathy, systemic sclerosis, lichen sclerosis)
Other: Morphea
Scleroderma, Localized, Morphea, Scleroderma, Circumscribed, Frontal Linear Scleroderma en Coup de Sabre, Scleroderma, Linear, Other Skin
Children’s Health
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