Search Results
within category "Immune System Disorders "
Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.
Efficacy and Safety of Viaskin Milk in Children With IgE-Mediated Cow's Milk Allergy (MILES)
The objectives of this study are to evaluate the safety and efficacy of Viaskin Milk after 12
months of epicutaneous immunotherapy (EPIT) treatment, for desensitizing IgE-mediated cow's
milk allergic children and to assess the long-term safety and therapeutic benefit with
Viaskin Milk.
Call 214-648-5005 studyfinder@utsouthwestern.edu
John Bird
108478
All
2 Years to 17 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT02223182
STU 092014-046
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Eligibility criteria for study enrollment:
Inclusion Criteria:
• Signed Informed Consent Form (ICF) by parent(s)/guardian(s) of subjects and informed
assent form (IAF) for subjects ≥7 years, or as per local or country specific
guidelines or regulations.
• Male or female subjects 2 to 17 years old at Visit 1.
• Documented medical history or physician-confirmed diagnosis of IgE-mediated CMA with
systemic symptoms related to ingestion of milk or dairy products.
• Subjects currently following a strict cow's milk-free diet, with no consumption of
dairy or baked milk products.
• Cow's milk-specific IgE level at screening ≥10 kU/L
• Positive Skin Prick Test (SPT) to cow's milk with a largest wheal diameter ≥6 mm.
• Positive DBPCFC at screening with an eliciting dose ≤300 mg cow's milk proteins
(approximately ≤9.4 mL of cow's milk).
• Negative urine pregnancy test for female subjects of childbearing potential. Female
subjects of childbearing potential must agree and commit to use effective medical
methods of contraception for the entire duration of their participation in the study.
Sexual abstinence will be accepted as an effective method of contraception for girls
below 15 years of age.
• Ability to perform spirometry procedures in accordance with the American Thoracic
Society guidelines (2005) for subjects ≥6 years old. Ability to perform peak
expiratory flow (PEF) measurements for subjects ≥5 years old. Subjects <8 years of age
who have documented inability to adequately perform spirometry can perform only the
PEF evaluation. Subjects <5 years of age may be enrolled if they had no clinical
features of moderate or severe persistent asthma severity (as defined by the 2007
National Heart, Lung, and Blood Institute [NHLBI] Guidelines) within 1 year before
Visit 1.
• Subjects and/or parents/guardians willing to comply with all study requirements during
participation in the study.
Exclusion Criteria:
• History of severe anaphylaxis to cow's milk resulting in hypotension, hypoxia or
neurological compromise (collapse, loss of consciousness or incontinence) or requiring
mechanical ventilation.
• Pregnancy or lactation.
• Spirometry forced expiratory volume in 1 second (FEV1) <80% of the predicted value at
Visit 1 for subjects ≥6 years and able to perform the spirometry, or PEF <80% of
predicted value at Visit 1 for subjects performing only the PEF measurements.
• Any clinical features of moderate or severe persistent asthma severity (as defined by
the 2007 NHLBI guidelines) and high daily doses of inhaled corticosteroids.
• Known allergy to the Viaskin patch materials or excipients, or to any of the
components of the food challenge formulas other than the cow's milk proteins.
• Allergy or known history of reaction to Tegaderm® medical dressing with no possibility
to use an alternative adhesive dressing authorized by the sponsor in replacement.
• Subjects having objective symptoms to the placebo formula leading to stopping the
challenge during the screening DBPCFC.
• Severe reaction during the screening DBPCFC defined as need for intubation, and/or
hypotension persisting after epinephrine administration, and/or the need for >2 doses
of epinephrine.
• Symptomatic allergy to pollens with symptoms during the pollen season that might
interfere with the symptoms observed during the DBPCFC, if the DBPCFC is performed
during the pollen season. Screening of such subjects should be made out of the pollen
season.
• Inability to discontinue short-acting antihistamines for 3 days or long-acting
antihistamines for 5 to 7 days (depending on the half-life) before the DBPCFC.
• Use of systemic long-acting corticosteroids within 12 weeks before Visit 1 and/or use
of systemic short-acting corticosteroids within 4 weeks before Visit 1 or use of
systemic long-acting or short-acting corticosteroids during screening (unless used to
treat symptoms triggered by the DBPCFC or triggered by accidental allergen
consumption; in the latter case DBPCFC must then be scheduled after a minimum of 7
wash-out days).
• Subjects with asthma conditions meeting 1 or several criteria below:
• Uncontrolled persistent asthma (as defined by the 2007 NHLBI guidelines) or
subject being treated with a combination therapy of medium or high daily dose of
inhaled corticosteroid with a long acting inhaled β2-agonist. Intermittent
asthmatic subjects who require intermittent use of inhaled corticosteroids for
rescue are permitted.
• At least 2 systemic corticosteroid courses for asthma within 1 year before Visit
1 or 1 oral corticosteroid course for asthma within 3 months before Visit 1, or
during screening (unless used to treat symptoms triggered by the DBPCFC).
• Prior intubation/mechanical ventilation due to asthma within 2 years before Visit
1, or during screening.
• Upper respiratory infection or gastroenteritis within 7 days of DBPCFC (DBPCFC must
then be rescheduled at least 7 days after resolution of these conditions).
• Any history of milk immunotherapy (eg, oral immunotherapy, sublingual immunotherapy or
specific oral tolerance induction).
• Prior history of any other food allergen immunotherapy (eg, oral immunotherapy,
sublingual immunotherapy or specific oral tolerance induction) within 5 years before
Visit 1.
• Subjects currently under aeroallergen immunotherapy and unwilling or unable to
discontinue at the time of Visit 1. Aeroallergen Immunotherapy must be discontinued at
the time of Visit 1.
• Use of any anti-IgE drug (eg, omalizumab), any immunomodulatory therapy, or any
biological agent therapy (eg, anti-tumor necrosis factor drugs) within 1 year before
Visit 1, or during screening.
• Generalized dermatologic diseases (eg, severe atopic dermatitis, uncontrolled
generalized eczema, icthyosis vulgaris) with no intact zones to apply the Viaskin
patch, or urticarial and mast cells disorders such as chronic idiopathic urticaria.
• Subject and/or subject's parents/guardians with obvious excessive anxiety and unlikely
to cope with the conditions of a food challenge.
• Past or current disease, including but not limited to active eosinophilic
gastrointestinal disorders, autoimmune disorders, immunodeficiency, malignancy,
uncontrolled disease (hypertension, diabetes, psychiatric disorder, cardiac disease),
or other disorders (eg, liver, gastrointestinal, kidney, cardiovascular, pulmonary
disease or blood disorder) which in the opinion of the Investigator or the sponsor may
affect the subject's participation in the study or place the subject at increased
risk.
• Subjects and/or parents/guardians unable to use the epinephrine auto-injector properly
in spite of being adequately trained.
• Contraindicated condition for the use of epinephrine.
• Use of any investigational drug or device, or participation in another interventional
clinical study within 3 months before Visit 1.
• Subjects receiving beta-blockers or Angiotensin converting-enzyme (ACE) inhibitors.
• Subjects unable to follow the protocol requirements.
An Investigational Study to Evaluate BMS-986165 in Patients With Systemic Lupus Erythematosus
This study will investigate BMS-986165 to assess its effects in patients with systemic lupus
erythematosus (SLE).
Call 214-648-5005 studyfinder@utsouthwestern.edu
David Karp
13762
All
18 Years to 75 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03252587
STU 062018-059
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For more information regarding Bristol-Myers Squibb Clinical Trial participation, please
visit www.BMSStudyConnect.com
Inclusion Criteria:
• Meets the Systemic Lupus International Collaborating Clinics (SLICC) classification
criteria for SLE and diagnosed ≥ 24 weeks before the screening visit
• One of the following: antinuclear antibody (ANA) ≥ 1:80 or positive
anti-double-stranded DNA (dsDNA) or positive anti-Smith (Sm)
• Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score ≥ 6 points
and clinical SLEDAI-2K score ≥ 4 points
Exclusion Criteria:
• Subjects with drug-induced SLE, certain other autoimmune diseases, and active, severe
lupus nephritis
• SLE overlap syndromes such as scleroderma and mixed connective tissue disease
• Clinically significant abnormalities on chest x-ray or ECG
• History of any significant drug allergy
Other protocol defined inclusion/exclusion criteria could apply
A Study of RVT-1401 in Myasthenia Gravis (MG) Patients
The purpose of the current study is to assess safety/tolerability and key pharmacodynamic
(PD) effects that are considered to be associated with clinical benefit (reduction of total
IgG and anti-AChR-IgG) in Myasthenia Gravis patients following treatment with RVT-1401 (also
known as IMVT-1401) compared to placebo.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Shaida Khan
137363
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT03863080
STU-2019-0635
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Inclusion Criteria:
1. Male or female ≥ 18 years of age.
2. Myasthenia Gravis Foundation of America (MGFA) Class II-IVa and likely not in need of
a respirator for the duration of the study as judged by the Investigator.
3. QMG score ≥12 at Screening and Baseline.
Other, more specific inclusion criteria are defined in the protocol.
Exclusion Criteria:
1. Use of rituximab, belimumab, eculizumab or any monoclonal antibody for
immunomodulation within 6 months prior to first dosing.
2. Immunoglobulins given by SC, IV (IVIG), or intramuscular route, or
plasmapheresis/plasma exchange (PE) within 4 weeks before Screening.
3. Thymectomy performed < 12 months prior to screening.
4. Total IgG level <6 g/L (at screening).
5. Absolute neutrophil count <1500 cells/mm3(at screening).
Other, more specific exclusion criteria are defined in the protocol
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.
Call 214-648-5005 studyfinder@utsouthwestern.edu
David Karp
13762
All
15 Years to 45 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.
Safety Study of Viaskin Peanut to Treat Peanut Allergy (REALISE)
This study evaluates the safety of Viaskin Peanut 250 mcg in the treatment of peanut allergy
in children from 4 to 11 years of age. Subjects will receive either Viaskin Peanut 250 mcg or
a placebo for a period of 6 months, after which all subjects will be receiving the active
treatment up to a period of 3 years under active treatment.
Call 214-648-5005 studyfinder@utsouthwestern.edu
John Bird
108478
All
4 Years to 11 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT02916446
STU 082016-034
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Inclusion Criteria:
• Physician-diagnosed peanut allergy;
• A peanut Skin Prick Test (SPT) with a wheal largest diameter ≥8 mm;
• A specific-peanut Immunoglobulin E (IgE) ≥14 kU/L;
• Subjects following a strict peanut-free diet.
Exclusion Criteria:
• Generalized dermatologic disease
• Spirometry forced expiratory volume in 1 second (FEV1) <80% of the predicted value, or
peak expiratory flow (PEF) <80% of predicted value;
• Receiving β-blocking agents, angiotensin-converting enzyme inhibitors,
angiotensin-receptor blockers, calcium channel blockers or tricyclic antidepressant
therapy; anti-tumor necrosis factor drugs or anti-IgE drugs (such as omalizumab) or
any biologic immunomodulatory therapy;
• Prior or concomitant history of any immunotherapy to any food allergy (for example
EPIT, OIT, SLIT, or specific oral tolerance induction).
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
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
David Sutcliffe
53153
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.
FARE Peanut SLIT and Early Tolerance Induction (FARE/SLIT)
Primary Objective: To determine if 36 months of peanut SLIT as an early intervention in
subjects ages 1 to 4 years induces clinical desensitization. The primary outcome of this
objective will be a statistically significant difference in challenge scores between the
treatment group versus the placebo group during DBPCFC (Double blind placebo controlled food
challenge) performed after 36 months of peanut SLIT (desensitization). A secondary outcome of
this objective will be a statistically significant difference in the challenge score of the
treatment group versus the placebo group during the DBPCFC performed 3 months after
discontinuing therapy (tolerance). Challenge scores are measured by the amount of peanut
protein participants are able to ingest successfully without symptoms of an allergic
reaction. [Time Frame: Baseline, 39 months]
Secondary Objective: To examine the change in immune parameters associated with peanut SLIT
and the development of clinical tolerance. Through this objective, the investigators will
seek to understand the molecular processes by which SLIT affects the immune system through
evaluation of immune mechanisms in relationship to clinical findings of desensitization and
tolerance. The investigators will delineate the impact of peanut SLIT on the subsequent
cellular and humoral responses to peanut protein: 1) peanut specific IgE, IgG, and IgG4
response, 2) peanut specific basophil activation, 3) mast cell responses through skin prick
testing, and 4) specific T-cell cytokine responses and T regulatory cell (TReg) activation.
The investigators anticipate that the effect of peanut SLIT will occur by induction of TRegs,
conversion of T cells from an allergic (TH2) to a non-allergic (TH1) lymphocyte response
(measured by cytokines, antibody levels, and skin prick test size), a change in
peanut-specific basophil activation, or through a combination of the above.
[Time Frame: Baseline, 39 months]
Call 214-648-5005 studyfinder@utsouthwestern.edu
John Bird
108478
All
12 Months to 48 Months old
Phase 2
This study is NOT accepting healthy volunteers
NCT02304991
STU 022014-047
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Inclusion Criteria:
• Written informed consent from participant's parent/guardian.
• Age 12-48 months of either sex, any race, any ethnicity.
• A peanut allergy diagnosis with a convincing clinical history of peanut allergy and a
serum peanut-specific IgE [UniCAP] > 0.35 kUA/L AND a positive skin prick test to
peanut (>3 mm than the negative control) OR are sensitized to peanut (based on a serum
IgE [UniCAP] to peanut of > 5 kUA/L) AND a positive skin prick test to peanut (> 3 mm
than the negative control) and no known history of ingestion of peanut.
• A positive DBPCFC to 1000 mg of peanut at enrollment.
Exclusion Criteria:
• History of severe anaphylaxis to peanut, defined as hypoxia, hypotension, or
neurologic compromise (cyanosis or SpO2 < 92% at any stage, hypotension, confusion,
collapse or loss of consciousness).
• Participation in any interventional study for the treatment of food allergy in the
past 6 months.
• Known oat, wheat, or glycerin allergy.
• Eosinophilic or other inflammatory (e.g. celiac) gastrointestinal disease.
• Severe asthma (2007 NHLBI Criteria Steps 5 or 6 •Appendix 2).
• Inability to discontinue antihistamines for skin testing and DBPCFCs.
• Use of omalizumab or other non-traditional forms of allergen immunotherapy (e.g., oral
or sublingual) or immunomodulator therapy (not including corticosteroids) or biologic
therapy within the past year.
• Use of beta-blockers (oral), angiotensin-converting enzyme (ACE) inhibitors,
angiotensin-receptor blockers (ARB) or calcium channel blockers.
• Significant medical condition (e.g., liver, kidney, gastrointestinal, cardiovascular,
hematologic, or pulmonary disease) which would make the subject unsuitable for
induction of food reactions.
Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis
The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with
generalized Myasthenia Gravis (gMG). Subjects will be randomized in a 1:1:1 ratio to receive
daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 weeks.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Shaida Khan
137363
All
18 Years to 85 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03315130
STU 102017-046
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Inclusion Criteria:
• Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IVa] at
Screening
• Positive serology for acetylcholine receptor (AChR) autoantibodies
• QMG score ≥ 12 at Screening and Randomization
• No change in corticosteroid dose for at least 30 days prior to Randomization or
anticipated to occur during the 12-week Treatment Period
• No change in immunosuppressive therapy, including dose, for at least 30 days prior to
Randomization or anticipated to occur during the 12-week Treatment Period
Exclusion Criteria:
• Thymectomy within 6 months prior to Randomization or scheduled to occur during the 12
week Treatment Period
• History of meningococcal disease
• Current or recent systemic infection within 2 weeks prior to Randomization or
infection requiring intravenous (IV) antibiotics within 4 weeks prior to Randomization
A Study of AeroVanc for the Treatment of MRSA Infection in CF Patients
This study is a multi-center, randomized phase III study to evaluate the clinical
effectiveness of AeroVanc in persistent MRSA in patients with Cystic Fibrosis.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Preeti Sharma
117060
All
6 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03181932
STU 092016-033
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Inclusion Criteria
1. Subjects ≥ 6 years of age at time of Informed Consent Form (ICF) or Assent Form
signing.
2. Confirmed diagnosis of CF, determined by having clinical features consistent with the
CF phenotype, plus one of the following:
1. Positive sweat chloride test (value ≥ 60 mEq/L),
2. Genotype with 2 mutations consistent with CF (i.e., a mutation in each of the
cystic fibrosis transmembrane conductance regulator [CFTR] genes).
3. Positive sputum culture or a throat swab culture for MRSA at Screening.
4. In addition to the Screening sample, have at least 2 prior sputum or throat swab
cultures positive for MRSA, of which at least 1 sample is more than 6 months prior to
Screening. At least 50% of all MRSA cultures (sputum or throat swab culture) collected
from the time of the first positive culture (in the previous 1 year) must have tested
positive for MRSA. (Note: Screening sample may count towards 50% positive count)
5. Forced expiratory volume in 1 second (FEV1) ≥ 30% and ≤ 90% of predicted that is
normal for age, gender, race, and height, using the Global Lung Function Initiative
(GLI) equation.
6. At least 1 episode of acute pulmonary infection treated with non-maintenance
antibiotics within 12 months prior to the Baseline visit. (Initiation of treatment
with intermittent inhaled anti-Pseudomonal therapy will not qualify as treatment with
non-maintenance antibiotics).
7. If female of childbearing potential, an acceptable method of contraception must be
used during the study and must be combined with a negative pregnancy test obtained
during Screening; sexually active male subjects of reproductive potential who are
non-sterile (i.e., male who has not been sterilized by vasectomy for at least 6
months, and were not diagnosed with infertility through demonstration of azoospermia
in a semen sample and/or absence of vas deferens through ultrasound) must be willing
to use a barrier method of contraception, or their female partner must use an
acceptable method of contraception, during the study.
For purposes of this study, the Sponsor defines "acceptable methods of contraception"
as:
1. Oral birth control pills administered for at least 1 monthly cycle prior to
administration of the study drug.
2. A synthetic progestin implanted rod (eg, Implanon®) for at least 1 monthly cycle
prior to the study drug administration but not beyond the 4th successive year
following insertion.
3. Intrauterine devices (IUDs), inserted by a qualified clinician for at least 1
monthly cycle prior to study drug administration.
4. Medroxyprogesterone acetate (eg, Depo-Provera®) administered for a minimum of 1
monthly cycle prior to administration of the study drug and continuing through 1
month following study completion.
5. Hysterectomy or surgical sterilization.
6. Abstinence.
7. Double barrier method (diaphragm with spermicidal gel or condoms with
contraceptive foam).
NOTE: For subjects prescribed Orkambi: Orkambi may substantially decrease hormonal
contraceptive exposure, reducing the effectiveness and increasing the incidence of
menstruation-associated adverse reactions. Hormonal contraceptives, including oral,
injectable, transdermal, and implantable, should not be relied upon as an effective
method of contraception when co-administered with Orkambi.
8. Able and willing to comply with the protocol, including availability for all scheduled
study visits and able to perform all techniques necessary to use the AeroVanc inhaler
and measure lung function.
9. Agree not to smoke during any part of the clinical trial (Screening visit through end
of study).
10. Subjects with a P. aeruginosa co-infection must either be stable on a regular
suppression regimen of inhaled antibiotics or must be, in the opinion of the
Investigator, stable despite the lack of such treatment.
Exclusion Criteria
1. Use of anti-MRSA treatments prescribed as maintenance therapy (intravenous [IV] or
inhaled treatment within 28 days; oral treatment within 14 days) prior to the Baseline
visit.
2. Use of non-maintenance antibiotic for pulmonary infection or extrapulmonary MRSA
infection (IV or inhaled antibiotic within 28 days; oral antibiotic within 14 days)
prior to the Baseline visit.
3. History of previous allergies or sensitivity to vancomycin, or other component(s) of
the study drug or placebo except for a history of red-man syndrome.
4. Inability to tolerate inhaled products.
5. First time sputum culture or throat swab culture yielding B. cepacia, or
nontuberculous Mycobacteria in the previous 6 months to Screening.
6. History of lung or other solid organ transplantation or currently on the list to
receive lung or other solid organ transplantation.
7. Resistance to vancomycin at Screening (vancomycin resistant Staphylococcus aureus
[VRSA], or vancomycin intermediate resistant Staphylococcus aureus [VISA], with
minimum inhibitory concentration [MIC] ≥ 8 μg/mL).
8. Oral corticosteroids in doses exceeding 10 mg prednisone per day or 20 mg prednisone
every other day, or equipotent doses of other corticosteroids.
9. Changes in antimicrobial, bronchodilator, anti-inflammatory or corticosteroid
medications within 14 days, or changes in CFTR modulators within 28 days, prior to the
Baseline visit.
10. Abnormal laboratory findings or other findings or medical history at Screening that,
in the Investigator's opinion, would compromise the safety of the subject or the
quality of the study data.
11. Inability to tolerate inhalation of a short acting beta2 agonist
12. SpO2 <90% at Screening.
13. Changes in physiotherapy technique or physiotherapy scheduled within 1 week of the
Baseline visit.
14. Administration of any investigational drug or device within 4 weeks prior to the
Screening visit and during the study
15. Female with positive pregnancy test result during Screening, pregnant (or intends to
become pregnant), lactating or intends to breastfeed during the study.
16. Renal insufficiency, defined as creatinine clearance < 50 mL/min using the
Cockcroft-Gault equation for adults or Schwartz equation for children at the Screening
visit.
17. Abnormal liver function, defined as ≥ 4x upper limit of normal (ULN), of serum
aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT), or known
cirrhosis at Screening.
18. Diagnosed with clinically significant hearing loss.
19. History of positive result for human immunodeficiency virus (HIV), hepatitis B virus
(HBV) or hepatitis C virus (HCV).
20. Planned hospitalizations for prophylaxis antibiotic treatment within 28 days prior to
Baseline visit or during the double-blind period (Period 1).
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.
Dynamic Imaging of Variation in Lupus Nephritis (DIVINE)
To use a variety of renal imaging modalities, including diffusion weighted imaging (DWI),
blood oxygen level dependent (BOLD) imaging, T1rho (T1rho) imaging, and dynamic contrast
enhanced magnetic resonance imaging (DCE-MRI) to evaluate the intra-renal blood flow,
perfusion, cellularity, fibrosis and atrophy within the kidneys of patients with lupus
nephritis (LN) and compare these parameters to renal biopsy findings to determine whether
DWI, BOLD, T1rho, and DCE-MRI may provide a set of non-invasive tools to assess renal
function and pathology in LN.
Call 214-648-5005 studyfinder@utsouthwestern.edu
David Karp
13762
All
18 Years to 65 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03180021
STU 032017-069
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Inclusion Criteria:
1. Provide written informed consent agreeing to all study procedures, before any study-
specific procedures are done.
2. Male and female subjects 18 to 65 years of age, inclusive.
3. Subjects currently being evaluated for new or recurrent LN with a SOC kidney biopsy
planned OR being evaluated for IgA nephropathy and with a SOC kidney biopsy planned.
4. Patients with LN must meet American College of Rheumatology (ACR) or Systemic Lupus
Collaborating Clinics (SLICC) criteria for Systemic Lupus Erythematosus (SLE).
5. Subjects with a life expectancy >6 months.
Exclusion Criteria:
1. Participation in another investigational study during same time period.
2. Contraindication to receiving a GBCA.
3. More than 2 previous lifetime exposures to a GBCA.
4. Any contraindication to MRI, including metal implants, claustrophobia or morbid
obesity.
5. Acute or chronic severe renal insufficiency (glomerular filtration rate [GFR] <40 mL
per minute per 1.73 m2).
6. Subject requiring dialysis.
7. Presence of pre-existing renal disease unrelated to SLE or IgA nephropathy,
respectively.
8. Acute renal insufficiency of any severity caused by the hepato-renal syndrome.
9. Previous or pre-existing nephrogenic systemic fibrosis.
10. History of clinically significant anti-phospholipid syndrome.
11. Chronic liver function impairment, indicated by liver function tests (aspartate
aminotransferase [AST], alanine aminotransferase [ALT]) >2-fold upper limit of normal.
12. Platelet count <50,000/μL.
13. Hemoglobin <8.0 g/dL.
14. History of or presence of central nervous system (CNS) disease such as active lupus
cerebritis or multiple sclerosis that might compromise blood brain barrier function.
15. Infection that is clinically relevant, particularly hepatitis B virus (HBV), hepatitis
C virus (HCV) and/or human immunodeficiency virus (HIV).
16. Pregnant or nursing females, or females not using effective contraception.
17. Inability or unwillingness to return to the research site clinic for study visits at
baseline and at 6 months.
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.
• 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: 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: 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: 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 12 weeks (84 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 iobenguane
(MIBG) positive (+) evaluable are eligible; measurable disease in patients with CNS
involvement is defined as tumor that is measurable in two perpendicular diameters on
magnetic resonance imaging (MRI) and visible on more than one slice
• 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: 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, 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) >= 70ml/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
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;
Inclusion Criteria
Each prospective subject must meet ALL of the following inclusion criteria in order to be
eligible for this study:
1. Signed written informed consent granted prior to initiation of any study-specific
procedures.
2. 18 years of age and older.
3. For the dose escalation cohorts, relapsed or refractory subjects with a diagnosis of
B-cell NHL, CLL/SLL and WM who have received at least two prior systemic therapies .
Subjects must have failed or are intolerant to standard therapies and cannot be a
candidate for standard salvage regimens. Subjects with low grade lymphoma must be
progressing and requiring treatment..
4. For the expansion cohorts, the following criteria must be met:
• Cohort A: Relapsed/Refractory (R/R) CLL/SLL subjects with at least 2 prior
systemic therapies and previously treated with a covalent BTKi who must have a
documented BTK mutation on C481 residue
• Cohort B: R/R CLL/SLL subjects who have failed or were intolerant to a BTKi with
documentation of the absence of BTK mutation on C481 residue. In this study,
intolerance to standard therapy is defined as having experienced a grade 3 or
higher adverse event that was caused by the standard therapy and resulted in
treatment discontinuation.
• Cohort C: Richter's transformation subjects who have failed at least one prior
therapy
• Cohort D: Follicular Lymphoma (FL) subjects who have failed at least 2 prior
systemic therapies and are histology grade 1, 2, or 3A
• Cohort E: Mantle Cell Lymphoma (MCL) subjects who have failed at least 2 prior
systemic therapies
• Cohort F: Marginal Zone Lymphoma (MZL) subjects who have failed at least 2 prior
systemic therapies
• Cohort G: High-grade B-cell lymphoma subjects who have failed at least 2 prior
systemic therapies and have known MYC and BCL2 and/or BCL6 translocations
• Cohort H: Waldenström macroglobulinemia (WM) subjects who have failed at least 2
prior systemic therapies
5. Disease status requirement:
1. For CLL subjects, symptomatic disease that mandates treatment (Hallek et al.
2018).
2. For B-cell NHL subjects, measurable disease by imaging scan.
3. For WM, serum immunoglobulin M (IgM) with a minimum IgM level of ≥ 2 times the
upper limit of normal (ULN).
6. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
7. Good organ function
1. Creatinine clearance of ≥ 60 mL/min as estimated by the Cockcroft-Gault equation
or by 24-hour urine collection.
2. Total bilirubin ≤ 1.5 x institutional ULN (total bilirubin of ≤ 3 x institutional
ULN in subjects with documented Gilbert's syndrome).
3. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 ×
institutional ULN.
4. Platelet count ≥ 50,000/µL
5. Absolute neutrophil count (ANC) ≥ 1000/µL.
6. Hemoglobin (Hgb) ≥ 8.0 g/dL, stable for ≥ 1 week.
8. For men and women of child-bearing potential, willing to use adequate contraception
(e.g., latex condom, cervical cap, diaphragm, abstinence, etc.) for the entire
duration of the study.
9. Female subjects of child-bearing potential must have a negative serum pregnancy test
within 14 days of the first day of drug dosing.
10. Ability to swallow oral medications without difficulty.
Exclusion Criteria
Potential subjects who meet ANY of the following exclusion criteria are not eligible
for enrollment into this study:
11. Had immunotherapy, radiotherapy, radioimmunotherapy, biological therapy, chemotherapy,
or treatment with an investigational product within 5 half-lives or four weeks
(whichever is shorter) prior to treatment initiation, or oral therapy within 5
half-lives or one week (whichever is shorter) prior to treatment initiation.
12. Transformation of FL to a more aggressive subtype of lymphoma or grade 3b FL
13. Subjects currently being treated with the following drugs:
1. CYP 2C9 substrates with a narrow therapeutic index (such as warfarin, phenytoin)
2. CYP 2C8 substrates with a narrow therapeutic index (such as paclitaxel)
3. CYP 2C19 substrates with a narrow therapeutic index (such as S-mephenytoin)
4. CYP 2D6 substrates with a narrow therapeutic index (such as thioridazine,
pimozide)
5. P-gp substrates with a narrow therapeutic index (such as digoxin) Note: A washout
period of at least 5 times the half-life after the last dose of any of the above
treatments is required for a subject to be eligible for study enrollment.
14. Prior allogeneic bone marrow transplant.
15. Active central nervous system (CNS) involvement.
16. Pregnant or breast-feeding women.
17. Has significant, ongoing co-morbid conditions which would preclude safe delivery of
the study drug.
18. Uncontrolled illness including but not limited to ongoing or active infection,
symptomatic congestive heart failure (New York Heart Association [NYHA] Class III or
IV heart failure), unstable angina pectoris, cardiac arrhythmia, cardiac infarction in
the past six months, and psychiatric illness that would limit compliance with study
requirements.
19. QTc prolongation (defined as a QTc > 450 msecs) or other significant electrocardiogram
(ECG) abnormalities including 2nd degree atrioventricular (AV) block type II, 3rd
degree AV block, or bradycardia (ventricular rate less than 50 beats/min). If the
screening ECG has a QTc > 450 msecs, the ECG can be submitted for a centralized,
cardiologic evaluation.
20. Active human immunodeficiency virus (HIV) infection, Hepatitis B, or Hepatitis C
infection.
21. Other medical or psychiatric illness or organ dysfunction which, in the opinion of the
Investigator, would either compromise the subject's safety or interfere with the
evaluation of the safety of the study agent.
22. History of prior cancer within < 1 year, except for basal cell or squamous cell
carcinoma of the skin, cervical cancer in situ or other in situ carcinomas.
Drug: ARQ 531, Drug: ARQ 531
Lymphoma, B-cell, Waldenström Macroglobulinemia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Follicular Lymphoma, Marginal Zone Lymphoma, Mantle Cell Lymphoma, Diffuse Large B Cell Lymphoma, Richter's Transformation
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
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
People with Primary Immune Deficiency (PID) may develop severe, life-threatening infections
as a result of inherited defects in the genes that normally instruct blood-forming cells to
develop and to fight infections. PID diseases include Severe Combined Immune Deficiency
(SCID), leaky SCID, Omenn syndrome (OS), and Reticular Dysgenesis (RD). PIDs may be treated
by transplantation of bone marrow stem cells from a healthy person or, in some cases, by
enzyme replacement or by gene therapy. Patients with SCID were among the first to receive
bone marrow stem cell (also called hematopoietic cells) transplantation (HCT) more than 40
years ago, and HCT is the standard treatment today for this group of diseases. Since PID
diseases are rare, there are not enough patients at any single center to determine the full
range of causes, natural history, or best methods of treatment. For this research study many
PID centers across North America have organized into the Primary Immune Deficiency Treatment
Consortium (PIDTC) to pool their experience and study PIDs together. Researchers will collect
information on your general health, psychological and developmental health, and the current
status of your immune system to help better define future approaches to PID treatments.
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.
Open Label Study of Subcutaneous Immunoglobulin (SCIg) in Myasthenia Gravis
The purpose of this study is to determine whether Hizentra is a safe and effective treatment
for people with myasthenia gravis (MG).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Jaya Trivedi
46764
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT02100969
STU 042015-029
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Inclusion Criteria:
• Must have MGFA MG grades 2, 3, or 4 generalized MG, according to the MGFA
classification system
• Elevated AChR or MuSK Ab
• Patient's signs and symptoms should not be better explained by another disease process
• IVIg maintenance dose of 0.2 to 2 gm/kg/4 weeks or equivalent dose administered every
2-4 weeks
• Stable IVIg for at least 3 cycles
• Able to complete the study and return for follow-up visits
• Able to give written informed consent before participating in the study
Exclusion Criteria:
• History of chronic degenerative, psychiatric, or neurologic disorder other than MG
that can produce weakness or fatigue
• Other major chronic or debilitating illnesses within six months prior to study entry
• Female patients who are premenopausal and are (a) pregnant, (b) breastfeeding, or (c)
not using an effective method of double barrier birth control
• Altered levels of consciousness, dementia, or abnormal mental status
• Thymectomy in the previous three months
• History of renal insufficiency or liver disease
• Skin disease that would interfere with assessment of injection site reaction
• History of severe reactions to IVIg or SCIg
• Participation in a research study within the last 3 months
• Treatment with rituximab or other biologics within 12 months of study entry
• Unable to provide informed consent
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
Sequential Natalizumab - Alemtuzumab Therapy in Patients With Relapsing Forms of Multiple Sclerosis (SUPPRESS)
The purpose of this study is to determine if a sequential combination therapy of natalizumab
and alemtuzumab induces peripheral tolerance and reduces the annualized relapse rate (ARR) in
patients with relapsing-remitting multiple sclerosis (RRMS).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Olaf Stuve
58631
All
18 Years to 60 Years old
Phase 4
This study is NOT accepting healthy volunteers
NCT03135249
STU 112016-060
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Inclusion Criteria:
1. Age between 18 and 60 years, inclusive.
2. Diagnosis of relapsing forms of MS using revised McDonald Criteria1.
3. Expanded Disability Status Scale (EDSS) 0 •5.5 (note: 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 monthly doses of continuous natalizumab therapy (300 mg/d).
5. Understands English, and gives informed consent.
Exclusion Criteria:
1. Natalizumab failure based on clinician's discretion.
2. Any prior exposure to alemtuzumab.
3. Progressive MS.
4. A diagnosis of Progressive multifocal leukoencephalopathy (PML).
5. Known hypersensitivity to alemtuzumab.
6. Initiation of new immunosuppressant treatment after the subject becomes
protocol-eligible (except for corticosteroids) or enrollment in a concurrent trial
with immuno-active pharmacotherapies.
7. Uncontrolled diabetes mellitus defined as HbA1c > 8% and/or requiring intensive
management.
8. History of cytopenia consistent with the diagnosis of myelodysplastic syndrome.
9. Clinically significant autoimmune disease other than MS that may affect the CNS,
including neuromyelitis optica (NMO), systemic lupus erythematosus (SLE), or Behcet
disease.
10. Active hepatitis B or C infection or evidence of cirrhosis.
11. HIV positivity.
12. Uncontrolled viral, fungal, or bacterial infection.
13. Positive pregnancy test or inability or unwillingness to use effective means of birth
control. Effective birth control is defined as:
1. Refraining from all acts of vaginal intercourse (abstinence),
2. Consistent use of birth control pills,
3. Tubal sterilization or male partner who has undergone vasectomy
4. Placement of intrauterine device
5. Use, with every act of intercourse, of a diaphragm with contraceptive jelly
and/or condoms with contraceptive foam.
14. Presence of metallic objects implanted in the body that would preclude the ability of
the subject to safely have MRI exams.
15. Psychiatric illness, mental deficiency, or cognitive dysfunction making compliance
with treatment or informed consent impossible.
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
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
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
This study is an access and distribution protocol for unlicensed cryopreserved cord blood
units (CBUs) in pediatric and adult patients with hematologic malignancies and other
indications.
• Disorders affecting the hematopoietic system that are inherited, acquired, or result
from myeloablative treatment
• Signed informed consent (and signed assent, if applicable) obtained prior to study
enrollment
• Pediatric and adult patients of any age
Exclusion Criteria:
• Patients who are receiving only licensed CBUs
• Cord blood transplant recipients at international transplant centers
• Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)
• Patients whose selected unlicensed CBU(s) will be more than minimally manipulated
Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders, Acute Myelogenous Leukemia (AML or ANLL), Acute Lymphoblastic Leukemia (ALL), Other Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases, Other Leukemia, Hodgkin Lymphoma, Non-hodgkin Lymphoma, Multiple Myeloma/ Plasma Cell Disorder (PCD), Inherited Abnormalities of Erythrocyte Differentiation or Function, Disorders of the Immune System, Automimmune Diseases, Severe Aplastic Anemia
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
Severe Combined Immunodeficiency (SCID), natural history study, SCID treatment
Study to Explore the Mechanism of Action of Ocrelizumab and B-Cell Biology in Participants With Relapsing Multiple Sclerosis (RMS) or Primary Progressive Multiple Sclerosis (PPMS)
This is an open-label, multicenter, biomarker study designed to be hypothesis-generating in
order to better understand the mechanism of action of ocrelizumab and B-cell biology in RMS
or PPMS. The study will be conducted in two cohorts i.e. RMS cohort (4 arm group) and PPMS
cohort (one arm group). RMS cohort: Ocrelizumab will be administered as two intravenous (IV)
infusions of 300 milligrams (mg) on Days 1 and 15. Subsequent doses will be given as single
600-mg infusions at Weeks 24 and 48. Participants will be randomized in 1:1:1 ratio to
receive lumbar puncture (LP) post-treatment at Week 12, 24, or 52 following the first dose of
ocrelizumab in three arm groups. A fourth RMS arm with delayed treatment start (Arm 4
[control group]) will not be a part of the randomization and will be recruited separately,
wherein treatment with ocrelizumab will be delayed for 12 weeks from pre-treatment baseline.
PPMS cohort: Ocrelizumab 600 mg will be administered as two 300-mg IV infusions separated by
14 days at a scheduled interval of every 24 weeks. Participants will receive a LP at the
start of the study before dosing with ocrelizumab and second LP at Week 52 following the
first dose of ocrelizumab. A long-term extension will be conducted for participants that
complete the study and continue to receive ocrelizumab, to be administered every 6 months
starting at Week 72 for up to 4 years.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Shanan Munoz
148781
All
18 Years to 55 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT02688985
STU 022016-068
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Inclusion Criteria:
General
Inclusion Criteria:
• For women of childbearing potential: agreement to remain abstinent (refrain from
heterosexual intercourse) or use contraceptive methods that result in a failure rate
of <1 percent (%) per year during the treatment period and for at least 24 weeks after
the last dose of study treatment or until their B-cells have repleted, whichever is
longer
Inclusion Criteria Specific to RMS Participants:
• Diagnosis of RMS in accordance with the 2010 revised McDonald criteria
• Expanded Disability Status Scale (EDSS) score of 0 to 5.5 points, inclusive, at
Screening
• Disease duration from the onset of multiple sclerosis symptoms less than (<) 15 years
in participants with an EDSS score greater than (>) 5.0 at Screening
• Either treatment-naive or receiving treatment with disease-modifying therapies,
including prior use of interferon (IFN)-beta-1a (Avonex®, Rebif®), IFN-beta-1b
(Betaseron®/Betaferon), or glatiramer acetate (Copaxone®).
• At least one clinically documented relapse in the past year and/or at least one
T1-weighted Gadolinium (Gd)-enhancing lesion in the past year and/or at least one new
T2 lesion in the past year at the time of enrollment
Inclusion Criteria Specific to RMS Cohort Arm 4 Participants:
• Must meet inclusion criteria for the RMS cohort
• Separate signed Informed Consent Form for the RMS Delayed Time to Start Control Arm
(Arm 4)
• Must be willing to remain on the same dose and regimen of current standard of care, or
no treatment if treatment-naïve, for 12 weeks after study enrollment The treating
and/or study physician must agree that the participant is eligible to remain on the
same dose and regimen of their current standard of care at Screening, or to receive no
treatment if the participant is treatment-naïve, for 12 weeks after study enrollment
Inclusion Criteria Specific to PPMS Participants:
• Diagnosis of PPMS in accordance with the 2010 revised McDonald criteria
• EDSS score of 3.0 •6.5 points, inclusive, at Screening
• Disease duration from the onset of multiple sclerosis symptoms <10 years in
participants with an EDSS at Screening less than or equal to (=) 5.0
• Documented history of either elevated immunoglobulin G (IgG) Index or one or more IgG
oligoclonal bands (OCBs) detected by isoelectric focusing
Exclusion Criteria:
• Diagnosis of secondary progressive multiple sclerosis without relapses for at least 1
year
• History or known presence of recurrent or chronic infection (human immunodeficiency
virus [HIV], syphilis, tuberculosis)
• History of cancer, including solid tumors and hematological malignancies (except basal
cell, in situ squamous cell carcinomas of the skin, and in situ carcinoma of the
cervix of the uterus that have been excised and resolved with documented clean margins
on pathology)
• Known presence or history of other neurologic disorders
• Contraindications or intolerance to oral or IV corticosteroids, including IV
methylprednisolone, according to the country label
• Contraindication for LP
• Previous treatment with B cell-targeted therapies (such as rituximab, ocrelizumab,
atacicept, belimumab, or ofatumumab)
• Previous treatment with natalizumab/Tysabri®, alemtuzumab, anti-CD4 agents,
cladribine, teriflunomide, cyclophosphamide, mitoxantrone, azathioprine, mycophenolate
mofetil, cyclosporine, methotrexate, total body irradiation, or bone marrow
transplantation
• Treatment with fingolimod/Gilenya®, dimethyl fumarate/Tecfidera®, or similar treatment
within 6 months prior to enrollment
• Systemic corticosteroid therapy within 4 weeks prior to Baseline
• Previous or concurrent treatment with any investigational agent or treatment with any
experimental procedure for multiple sclerosis (such as treatment for chronic
cerebrospinal venous insufficiency)
• Certain laboratory abnormalities or findings at Screening
• Pregnant or lactating, or intending to become pregnant during the study
Exclusion Criteria Specific to RMS Participants:
Diagnosis of PPMS or secondary progressive multiple sclerosis without relapses
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
Dimethyl Fumarate (DMF) Observational Study (ESTEEM)
The primary objective of the study is to determine the incidence, type, and pattern of
serious adverse events (SAEs), including but not limited to infections (including
opportunistic infections), hepatic events, malignancies, and renal events, and of adverse
events (AEs) leading to treatment discontinuation in patients with MS treated with dimethyl
fumarate (DMF). Secondary objectives of this study in this population are as follows: To
determine dimethyl fumarate (DMF) prescription and utilization patterns in routine clinical
practice in patients with multiple sclerosis (MS); To assess the effectiveness of dimethyl
fumarate (DMF) on multiple sclerosis (MS) disease activity and disability progression in
routine clinical practice as determined by the Expanded Disability Status Scale (EDSS) score
and multiple sclerosis (MS) relapse information; and To assess the effect of dimethyl
fumarate (DMF) on health-related quality of life, healthcare resource consumption, and work
productivity.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Darin Okuda
146752
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT02047097
STU 032017-026
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Key
Inclusion Criteria:
•Patients with multiple sclerosis (MS) who are newly initiating treatment with dimethyl
fumarate (DMF) under routine clinical care are eligible to participate in the study.
Key
Exclusion Criteria:
• Patients with previous exposure to dimethyl fumarate (DMF), Fumaderm (fumaric acid
esters), or compounded fumarates.
• Patients participating in other clinical studies.
NOTE: Other protocol-defined inclusion/exclusion criteria may apply.