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93 Study Matches

Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) Plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors

This is a Phase 1 study of the combination of two drugs: MM-398 and Cyclophosphamide. The goal is to find the highest dose of MM-398 that can be given safely when it is used together with the chemotherapy drug Cyclophosphamide.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Patrick Leavey
35610
All
12 Months to 20 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT02013336
STU 092013-007
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Inclusion Criteria:

• Histologically or cytologically-confirmed Ewing sarcoma, rhabdomyosarcoma, neuroblastoma, or osteosarcoma
• Disease progression after prior therapy in locally advanced or metastatic setting
• Measurable or evaluable disease based on the Response Evaluation Criteria in Solid Tumors (RECIST v1.1) criteria
• Age 12 months to <21 years
• Adequate bone marrow reserves, hepatic function, and renal function
• Recovered from effects of any prior surgery or cancer therapy
• Patients 18 years or older will provide written consent. A parent or legal guardian of a patient <18 years of age will provide informed consent and patients 11 to 18 years of age will provide written assent or as per participating institutional policy.
Exclusion Criteria:

• Clinically significant gastrointestinal disorders
• NYHA Class III or IV congestive heart failure, ventricular arrhythmias or uncontrolled blood pressure
• Active infection or unexplained fever
• Known hypersensitivity to any of the components of MM-398 or other liposomal products
• Recent Investigational therapy
• Pregnant or breast feeding; females of child-bearing potential must test negative for pregnancy at the time of enrollment
Drug: MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamide
Neuroblastoma, Sarcoma, Ewing Sarcoma, Mycosis Fungoides, Rhabdomyosarcoma, Osteosarcoma, Recurrent or Refractory Solid Tumors, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Hodgkins Lymphoma, Kaposis sarcoma, Other Hematopoietic, Small Intestine, Soft Tissue
pediatric, MM-398, cyclophosphamide, irinotecan
Children’s Health
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A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

This study will evaluate the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation
Call 214-648-5005
studyfinder@utsouthwestern.edu, ASHLEY.KELLER@UTSouthwestern.edu
Raksha Jain
19733
All
12 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03525574
STU 022018-086
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Inclusion Criteria:

• Completed study drug treatment in a parent study; or had study drug interruption(s) in a parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study.
Exclusion Criteria:

• History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator.
• Current participation in an investigational drug trial (other than a parent study) Other protocol defined Inclusion/Exclusion criteria may apply.
Drug: VX-445, Drug: TEZ, Drug: IVA, Drug: IVA
Cystic Fibrosis, Lung/Thoracic
UT Southwestern; Children’s Health
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Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del

This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in subjects 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).
Call 214-648-5005
studyfinder@utsouthwestern.edu, Mary.Klosterman@UTSouthwestern.edu
Preeti Sharma
117060
All
12 Months to 23 Months old
Phase 3
This study is NOT accepting healthy volunteers
NCT03601637
STU 052018-060
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Key
Inclusion Criteria:

• Subjects will be 1 to less than 2 years of age on Day 1 of the relevant part of the study.
• Homozygous for F508del (F/F). Key
Exclusion Criteria:

• Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject.
• Solid organ or hematological transplantation. Other protocol defined Inclusion/Exclusion criteria may apply.
Drug: LUM, Drug: IVA
Cystic Fibrosis
Children’s Health
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A Study of Selpercatinib (LOXO-292) in Participants With Advanced Solid Tumors, RET Fusion-Positive Solid Tumors, and Medullary Thyroid Cancer (LIBRETTO-001) (LIBRETTO-001)

This is an open-label, first-in-human study designed to evaluate the safety, tolerability, pharmacokinetics (PK) and preliminary anti-tumor activity of selpercatinib (also known as LOXO-292) administered orally to participants with advanced solid tumors, including rearranged during transfection (RET)-fusion-positive solid tumors, medullary thyroid cancer (MTC) and other tumors with RET activation.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Tian Zhang
206021
All
12 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT03157128
STU 082018-008
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Key
Inclusion Criteria:
For Phase 1:
• Participants with a locally advanced or metastatic solid tumor that:
• Has progressed on or is intolerant to standard therapy, or
• For which no standard therapy exists, or in the opinion of the Investigator, are not candidates for or would be unlikely to tolerate or derive significant clinical benefit from standard therapy, or
• Decline standard therapy
• Prior multikinase inhibitors (MKIs) with anti-RET activity are allowed
• A RET gene alteration is not required initially. Once adequate PK exposure is achieved, evidence of RET gene alteration in tumor and/or blood is required as identified through molecular assays, as performed for clinical evaluation
• Measurable or non-measurable disease as determined by RECIST 1.1 or RANO as appropriate to tumor type
• Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2 or Lansky Performance Score (LPS) greater than or equal to (≥) 40 percent (%) (age less than [<] 16 years) with no sudden deterioration 2 weeks prior to the first dose of study treatment
• Adequate hematologic, hepatic and renal function
• Life expectancy of at least 3 months For Phase 2: As for phase 1 with the following modifications:
• For Cohort 1: Participants must have received prior standard therapy appropriate for their tumor type and stage of disease, or in the opinion of the Investigator, would be unlikely to tolerate or derive clinical benefit from appropriate standard of care therapy
• Cohorts 1 and 2:
• Enrollment will be restricted to participants with evidence of a RET gene alteration in tumor
• At least one measurable lesion as defined by RECIST 1.1 or RANO, as appropriate to tumor type and not previously irradiated
• Cohorts 3 and 4: Enrollment closed
• Cohort 5:
• Cohorts 1-4 without measurable disease
• MCT not meeting the requirements for Cohorts 3 or 4
• MTC syndrome spectrum cancers (e.g., MTC, pheochromocytoma), cancers with neuroendocrine features/differentiation, or poorly differentiated thyroid cancers with other RET alteration/activation may be allowed with prior Sponsor approval
• cfDNA positive for a RET gene alteration not known to be present in a tumor sample
• Cohort 6: Participants who otherwise are eligible for Cohorts 1, 2 or 5 who discontinued another RET inhibitor may be eligible with prior Sponsor approval
• Cohort 7: Participants with a histologically confirmed stage IB-IIIA NSCLC and a RET fusion; determined to be medically operable and tumor deemed resectable by a thoracic surgical oncologist, without prior systemic treatment for NSCLC Key Exclusion Criteria (Phase 1 and Phase 2):
• Phase 2 Cohorts 1 and 2: an additional known oncogenic driver
• Cohorts 3 and 4: Enrollment closed
• Cohorts 1, 2 and 5: prior treatment with a selective RET inhibitor Notes: Participants otherwise eligible for Cohorts 1, 2, and 5 who discontinued another selective RET inhibitor may be eligible for Phase 2 Cohort 6 with prior Sponsor approval
• Investigational agent or anticancer therapy (including chemotherapy, biologic therapy, immunotherapy, anticancer Chinese medicine or other anticancer herbal remedy) within 5 half-lives or 2 weeks (whichever is shorter) prior to planned start of LOXO-292 (selpercatinib). In addition, no concurrent investigational anti-cancer therapy is permitted Note: Potential exception for this exclusion criterion will require a valid scientific justification and approval from the Sponsor
• Major surgery (excluding placement of vascular access) within 2 weeks prior to planned start of LOXO-292 (selpercatinib)
• Radiotherapy with a limited field of radiation for palliation within 1 week of planned start of LOXO-292 (selpercatinib), with the exception of participants receiving radiation to more than 30% of the bone marrow or with a wide field of radiation, which must be completed at least 4 weeks prior to the first dose of study treatment
• Any unresolved toxicities from prior therapy greater than Common Terminology Criteria for Adverse Events (CTCAE) Grade 1 at the time of starting study treatment with the exception of alopecia and Grade 2, prior platinum-therapy related neuropathy
• Symptomatic primary CNS tumor, metastases, leptomeningeal carcinomatosis, or untreated spinal cord compression. Participants are eligible if neurological symptoms and CNS imaging are stable and steroid dose is stable for 14 days prior to the first dose of LOXO-292 (selpercatinib) and no CNS surgery or radiation has been performed for 28 days, 14 days if stereotactic radiosurgery (SRS)
• Clinically significant active cardiovascular disease or history of myocardial infarction within 6 months prior to planned start of LOXO-292 (selpercatinib) or prolongation of the QT interval corrected (QTcF) greater than (>) 470 milliseconds (msec)
• Participants with implanted pacemakers may enter the study without meeting QTc criteria due to nonevaluable measurement if it is possible to monitor for QT changes.
• Participants with bundle branch block may be considered for study entry if QTc is appropriate by a formula other than Fridericia's and if it is possible to monitor for QT changes.
• Required treatment with certain strong cytochrome P450 3A4 (CYP3A4) inhibitors or inducers and certain prohibited concomitant medications
• Phase 2 Cohort 7 (neoadjuvant treatment): Participant must not have received prior systemic therapy for NSCLC.
Drug: LOXO-292
Lymphoma, Non-Small Cell Lung Cancer, Colon Cancer, Medullary Thyroid Cancer, Any Solid Tumor, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Esophagus, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Stomach, Thyroid, Urinary Bladder, Hodgkins Lymphoma, Small Intestine, Soft Tissue
LOXO-292, KIF5B-RET, M918T, CCDC6-RET, RET-PTC1, NCOA4-RET, RET-PTC, RET-PTC3, RET-PTC4, PRKAR1A-RET, RET-PTC2, GOLGA5-RET, RET-PTC5, ERC1-RET, KTN1-RET, RET-PTC8, HOOK3-RET, PCM1-RET, TRIM24-RET, RET-PTC6, TRIM27-RET, TRIM33-RET, RET-PTC7, AKAP13-RET, FKBP15-RET, SPECC1L-RET, TBL1XR1-RET, BCR-RET, FGRF1OP-RET, RFG8-RET, RET-PTC9, ACBD5-RET, MYH13-RET, CUX1-RET, KIAA1468-RET, FRMD4A-RET, SQSTM1-RET, AFAP1L2-RET, PPFIBP2-RET, EML4-RET, PARD3-RET, G533C, C609F, C609G, C609R, C609S, C609Y, C611F, C611G, C611S, C611Y, C611W, C618F, C618R, C618S, C620F, C620R, C620S, C630R, C630Y, D631Y, C634F, C634G, C634R, C634S, C634W, C634Y, K666E, E768D, L790F, V804L, V804M, A883F, S891A, R912P, CLIP1-RET, Y806C, RET fusion, RET alteration, RET mutation, RET rearrangement, RET translocation, Neoplasms by Site, Neoplasms, Non-Small Cell Lung Cancer, Lung Neoplasms, Carcinoma, Non-Small-Cell Lung, Cancer of Lung, Cancer of the Lung, Lung Cancer, Neoplasms, Lung, Neoplasms, Pulmonary, Pulmonary Cancer, Pulmonary Neoplasms, Respiratory Tract Neoplasms, Lung Diseases, Respiratory Tract Diseases, Carcinoma, Bronchogenic, Bronchial Neoplasms, Medullary Thyroid Cancer, Papillary Thyroid Cancer, Thyroid Diseases, Thyroid Neoplasms, Cancer of the Thyroid, Cancer of Thyroid, Neoplasms, Thyroid, Thyroid Ademona, Thyroid Cancer, Thyroid Carcinoma, Endocrine System Diseases, Endocrine Gland Neoplasms, Head and Neck Neoplasms, Thoracic Neoplasms, CNS tumor, Primary CNS tumor, Cancer of Colon, Cancer of the Colon, Colon Cancer, Colon Neoplasms, Colonic Cancer, Neoplasms, Colonic, Malignant tumor of Breast, Mammary Cancer, Mammary Carcinoma, Human, Mammary Neoplasm, Human, Neoplasms, Breast, Tumors, Breast, Human Mammary Carcinoma, Malignant Neoplasm of Breast, Breast Carcinoma, Breast Tumors, Cancer of the Breast, Breast Neoplasms, Breast Cancer, RET Inhibitor, MTC, NSCLC, selpercatinib, neo-adjuvant treatment in early stage NSCLC
UT Southwestern
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Markers of Osteoporosis in Cystic Fibrosis

Main Study Up to 100 subjects, both non-CF volunteers and Cystic Fibrosis (CF) patients, will participate in a single study visit that will include a DEXA scan, micro CT, and blood collection. Denosumab (Prolia) Sub study Approximately 10 adult subjects with CF who participated in the main study and have results indicating bone disease will receive treatment with Denosumab for up to 5 years. They will be asked to return annually for repeat DEXA scans, micro CT, and blood collection.
Call 214-648-5005
studyfinder@utsouthwestern.edu, YAMEI.CHENG@UTSouthwestern.edu
Raksha Jain
19733
All
18 Years to 64 Years old
Phase 4
This study is also accepting healthy volunteers
NCT03921060
STU 052018-007
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Cystic Fibrosis Main Study
Inclusion Criteria:

• Must have CF diagnosis confirmed by sweat test or genotype analysis
• Subjects (and parents/legal guardians as applicable) must have the ability to read and write in English Sub-study
Exclusion Criteria:

• No CF diagnosis
• Men or women without osteoporosis
• Less than 18 years of age
• Unwilling to return annually for study visits for up to 5 years
• Unwilling and/or medically unable to take denosumab
Drug: Denosumab
Cystic Fibrosis, Bones and Joints, Lung/Thoracic
UT Southwestern; Children’s Health
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Extracorporeal Photopheresis for Medicare Recipients of Lung Allografts (ECP)

The primary aims of this study is to determine the efficacy and tolerability of Extracorporeal Photopheresis (ECP) for the treatment of either refractory (240) or newly diagnosed (400-450) Bronchiolitis Obliterans Syndrome (BOS) in patients after lung transplantation.In compliance with the Centers for Medicare and Medicaid Services' (CMS) Coverage with Evidence Development (CED) decision, the study will collect specified demographic, comorbidity, treatment, and outcome data exclusively for Medicare beneficiaries who are treated with ECP for either refractory or New BOS.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Monika.Ruscheinsky@UTSouthwestern.edu
Amena Usmani
162809
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT02181257
STU-2018-0362
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INCLUSION Criteria for REFRACTORY BOS 1. Age (18 years old or older). 2. Medicare-eligible status 3. Lung transplant recipient (combined organ transplant recipients, e.g. heart-lung or liver-lung or lung re- transplantation recipients are eligible). 4. Patients with a diagnosis of BOS using at least two laboratory based FEV1 values obtained at least three weeks apart that are both at least 20% lower than baseline FEV1 using the International Society for Heart and Lung Transplantation (ISHLT) definition (The average of the two highest FEV1 measurements obtained at least 3 weeks apart after transplantation). The date of Diagnosis of New BOS is the first date of the two FEV1s that were used for the BOS diagnosis. 5. Refractory BOS defined as ongoing decline in FEV1 despite at least one of the following treatments: azithromycin, high-dose steroid, anti-thymocyte globulin, total lymphoid irradiation, sirolimus, or everolimus. 6. At minimum five recorded FEV1 measurements obtained at intervals of at least two weeks apart, over the 9 months preceding study enrollment, of which one FEV1 must be within two weeks prior to enrollment. 7. History of frequent spirometry monitoring defined as having had regular FEV1 measurements within the context of either of the following two options: (1) During the preceding four months prior to enrollment with no time interval between FEV1 measurements that exceeds 8 weeks. (2) During the preceding six months prior to enrollment with no time interval between FEV1 measurements that exceeds 12 weeks. 8. A documented clinical assessment including a physical assessment and Complete Blood Count (CBC) with White Blood Cell Count (WBC) within two weeks prior to enrollment. INCLUSION criteria for NEWLY Diagnosed BOS 1. Age (18 years old or older) 2. Medicare-eligible status. 3. Lung transplant recipient (combined organ transplant recipients, e.g. heart-lung or liver-lung, lung re-transplantation recipients, are eligible). 4. History of close FEV1 monitoring prior to diagnosis of new BOS defined as having had either of the two monitoring approaches: (1) Frequent laboratory based spirometry defined as having had regular FEV1 measurements within the context of either of the following two options: A. During the preceding six months prior to diagnosis of new BOS with no time interval between FEV1 measurements that exceeds 8 weeks. (Participants must be at least 6 months post transplant) B. During the preceding nine months prior to diagnosis of new BOS with no time interval between FEV1 measurements that exceeds 12 weeks (Participants must be at least 9 months post- transplant) (2) Frequent Home Spirometry through the separate IRB approved Standardized Home Spirometry Method sub-protocol. 5. Diagnosis of new BOS (i.e., "new BOS" is defined as within nine weeks of enrollment) based on laboratory-based spirometric FEV1 measurements obtained on at least two separate occasions (i.e., at least 3 weeks apart) that have declined by more than 20% from post-transplant baseline values (i.e., using ISHLT definition). The date of Diagnosis of New BOS is the first date of the two FEV1s that were used for the BOS diagnosis. Inherent to the diagnosis of new BOS is the exclusion of other potential causes of allograft dysfunction such as acute rejection, respiratory tract infection, and airway anastomotic complications. Thus, sites are encouraged to conduct appropriate evaluation for declining allograft function including bronchoscopy with bronchoalveolar lavage (BAL) and lung biopsies if clinically appropriate to exclude other potential causes of allograft dysfunction. 6. Achievement of a statistically significant rate of decline in lung function (FEV1) at the diagnosis of new BOS per the criteria in Section 3.6 as assessed by the following criteria: 1. For patients who are monitored with laboratory based spirometry, at least five recorded FEV1 measurements obtained at intervals of at least two weeks apart, over either the 6 or 9 (i.e., depending on the frequency of spirometry testing) months preceding study enrollment accompanied by a statistically significant (p<0.05) rate of decline of FEV1 that exceeds 30 mL/month; or 2. For patients who are monitored with home Spirometry, 4-6 recorded home spirometry FEV1 measurements obtained one week apart, over the 4-6 weeks prior to a confirmed FEV1 variance (i.e., the date of the second of two consecutive FEV1 values below the patient's normal range) along with 4-6 recorded weekly FEV1 measurements obtained after a confirmed variance accompanied by a statistically significant (p<0.05) rate of de-cline of FEV1 that exceeds 30 mL/month 7. Documented clinical assessment including a physical assessment and a CBC with WBC within two weeks prior to enrollment. EXCLUSION Criteria (Subjects meeting any one of these criteria will be excluded) 1. Current participation in another clinical treatment trial with an investigational agent used to manage BOS before or after enrollment. 2. Any condition that may interfere with the subject's ability to perform pulmonary function testing. 3. Known allergy or hypersensitivity to pharmacologic agents used during ECP 4. Any condition that would significantly affect the participant's ability to adhere to the protocol, affect interpretation of the study results, or put the participant at unacceptable risk for study-related complications as judged by the referring clinician. This may include a) patients with a specific acute contraindication to receiving ECP due to any acute condition such as new or evolving myocardial infarction or central nervous system disorder, hemodynamic instability or hypovolemia, acute bleeding, respiratory distress. 5. Patients with lupus erythematosus, porphyria cutanea tarda, erythropoietic protoporphyria, variegate porphyria, xeroderma pigmentosum, albinism, or other dermatologic or ocular condition that contraindicates the use of methoxsalen or markedly enhances photosensitivity in the investigator's judgment. 6. Aphakia or absence of ocular lenses 7. Pregnancy (positive pregnancy test
•a urine or blood pregnancy test must be obtained within 2 weeks prior to enrollment in women of childbearing potential) 8. Inability to provide informed consent or to comply with study treatments or assessments (e.g. due to cognitive impairment or geographic distance) 9. Recent (i.e., within 2 weeks prior to enrollment) leukopenia (white blood cell count < 30K/cumm or 3,000/mm3/ or 3.0 109 /L) 10. Patients whose decline in lung function (FEV1) is related to either Restrictive Chronic Lung Allograft Dysfunction (CLAD) or other causes that do not represent BOS such as pneumonia, heart failure, etc. For patients under review for eligibility for ECP for refractory BOS: 11. Patients with a post-transplant baseline FEV1 > 3 liters and most recent FEV1 < 900 mL 12. Patients with a post-transplant FEV1< 3 liters and the most recent FEV1 < 30% of post-transplant baseline 13. Rate of FEV1 decline within the last 6 or 9 months > 300 mL/month. 14. History of receiving ECP therapy within 6 months prior to enrollment. For patients under review for eligibility for RCT: 15. Patients post-transplant treated with any agent that depletes T lymphocytes for In-duction, acute cellular rejection or for any other reason can only be enrolled 12 months after the last dose of these agents assuming they meet enrollment inclusion criteria. T Lymphocyte depleting therapies include (but not limited to):
• monoclonal antibodies such as Alemtuzumab (Campath) that target CD52 T cell receptors
• polyclonal antibodies such as anti-thymocyte globulin (ATG) via immunization of rabbits (rATG) to either human thymocytes or Jurkat cells or via immunization of horses (hATG) to human thymocytes
• Radiation. Anti-B cell agents that do not deplete T lymphocytes such as Rituximab can be used and will not affect eligibility. 16. Any patient who at least 6 months after transplant is treated with an escalated dose of steroids (i.e., prednisone greater than 30 mg/day or that exceeds 900 mg in a 30 day period or equipotent doses of other steroids like Solumedrol ) for more than one month for an acute decline in lung function that is suspected to be secondary to acute cellular rejection.
Combination Product: Extracorporeal Photopheresis (ECP)
Bronchiolitis Obliterans Syndrome (BOS)
Bronchiolitis Obliterans Syndrome, Lung Transplantation, Extracorporeal Photopheresis, Methoxsalen
UT Southwestern
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LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With Langerhans Cell Histiocytosis

The LCH-IV is an international, multicenter, prospective clinical study for pediatric Langerhans Cell Histiocytosis LCH (age < 18 years).
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Erin Butler
104034
All
up to 18 Years old
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT02205762
STU-2018-0071
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Inclusion Criteria:

• Stratum I
• Patients must be less than 18 years of age at the time of diagnosis.
• Patients must have histological verification of the diagnosis of Langerhans cell histiocytosis according to the criteria described in Section 6.1
• Signed informed consent form
• Stratum II
• Patients of Stratum I who have:
• Progressive disease (AD worse) in non-risk organs after 6 weeks (Initial Course
• AD intermediate or worse in non-risk organs or AD better in risk organs after 12 weeks (Initial Course 2)
• Disease progression (AD worse) in non-risk organs at any time during continuation treatment
• Active disease at the end of Stratum I treatment
• Disease reactivation in non-risk organs at any time after completion of Stratum I treatment
• Stratum III
• Patients from Stratum I who fulfill the following criteria:
• AD worse in risk organs after week 6 (after Initial Course 1), or AD worse or AD intermediate in risk organs after week 12 (after Initial Course 2).
• Presence of unequivocally severe organ dysfunction at the above mentioned evaluation points (hematological dysfunction, liver dysfunction, or both of them) as
• Hb <70 g/L (<7.0 g/dl) and/or transfusion dependency
• PLT <20 x109/L (20,000/μL) and/or transfusion dependency (both criteria have to be fulfilled) AND/OR
• Liver dysfunction (or digestive involvement with protein loss)
• Total protein <55 g/L or substitution dependency
• Albumin <25 g/L or substitution dependency (at least one of the two criteria to be fulfilled)
• Stratum IV
• Patients from Stratum I or Stratum III who fulfill the following criteria:
• AD worse in risk organs after week 6 (after Initial Course 1), or AD worse or AD intermediate in risk organs after week 12 (after Initial Course 2) of Stratum I OR
• AD worse after the 2nd and 3rd 2-CdA/Ara-C course, and those AD worse or AD intermediate after the 4th 2-CdA/Ara-C course of Stratum III AND
• Presence of unequivocally severe organ dysfunction at the above mentioned evaluation points (hematological dysfunction, liver dysfunction, or both of them) as defined in Table XI (see Section 10.3.1).
• Informed consent: All patients or their legal guardians (if the patient is <18 years of age) must sign an Ethics or institutional Review Board approved consent form indicating their awareness of the investigational nature and the risks of this study. When appropriate, younger patients will be included in all discussions in order to obtain assent.
• Adequate organ function: Patients should have adequate hepatic, renal, cardiac and pulmonary function to undergo reduced intensity HCT based upon local institutional guidelines, or at a minimum meet requirements noted in eligibility checklist Appendix A-VIII_1. However, significant hepatic and pulmonary dysfunction, if secondary to underlying LCH disease activity, will not exclude patients from protocol enrollment and should be discussed with the National PI Coordinator and the Coordinating Principal Investigator.
• Stratum V
• All patients with verified diagnosis of LCH and MRI findings consistent with ND-CNSLCH irrespective of previous treatments (also those not registered to other Strata ofLCH-IV).
• Patients with isolated tumorous CNS-LCH (including isolated DI with mass lesion in the hypothalamus-pituitary axis). In patients with already established diagnosis of LCH and radiologic finding of CNS lesions compatible with LCH, a biopsy of the lesion is not obligatory. In all other cases a biopsy of the lesion is needed for inclusion into the study
• Stratum VI -- Patients with newly diagnosed SS-LCH and localization other than "multifocal bone",isolated tumorous CNS lesion, or isolated "CNS-risk" lesion.
• Stratum VII -- All patients registered in LCH IV (regardless of treatment) as long as consent for longterm follow-up has not been withheld.
Exclusion Criteria:

• Stratum I
• Pregnancy (patients of child-bearing age must be appropriately tested before chemotherapy)
• LCH-related permanent consequences (e.g. vertebra plana, sclerosing cholangitis, lung fibrosis, etc.) in the absence of active disease
• Prior systemic therapy
• Stratum II
• Patients with progressive disease in risk organs
• Permanent consequences (e.g. sclerosing cholangitis, lung fibrosis, etc.) without evidence of active LCH in the same organ or in any other locations
• No written consent of the patient or his/her parents or legal guardian
• Stratum III
• The presence of any of the following criteria will exclude the patient from the study:
• Isolated sclerosing cholangitis without evidence of active hepatic LCH as the only evidence of risk organ involvement.
• Inadequate renal function as defined by serum creatinine > 3x normal for age
• Stratum IV
• Pulmonary failure (requiring mechanical ventilation) not due to active LCH.
• Isolated liver sclerosis or pulmonary fibrosis, without active LCH.
• Uncontrolled active life-threatening infection.
• Decreased renal function with a GFR of less than 50ml/1.73m2/min.
• Pregnancy or active breast feeding
• Failure to provide signed informed consent
• Stratum VI
• Patients with SS-LCH who have an isolated tumorous CNS lesion (they are eligible for Stratum V),
• Patients with isolated "CNS-risk" or multifocal bone lesions (they are eligible for Stratum I, Group 2)
Drug: Prednisone, Drug: Vinblastine, Drug: mercaptopurine, Drug: INDOMETHACIN, Drug: Methotrexate, Drug: Cytosine Arabinoside, Drug: 2-chlorodeoxyadenosine, Procedure: hematopoietic stem cell transplantation (RIC-HSCT), Biological: Intravenous immunoglobulin
Langerhans Cell Histiocytosis, Brain and Nervous System, Bones and Joints, Liver, Lung/Thoracic, Other Hematopoietic
Langerhans cell histiocytosis
Children’s Health
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Evaluation of the Diagnostic Value of the TPD System in Determining ADHF Causing Acute Dyspnea

The objective of this study is to evaluate Lung Doppler signals (LDS) among patients presenting to the emergency department with acute dyspnea, in order to determine the diagnostic value of this non-invasive method to discriminate ADHF causing dyspnea from any other cause i.e., non-ADHF causes of dyspnea.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Riley.Martin@UTSouthwestern.edu
Deborah Diercks
152662
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT03998410
STU-2019-0989
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Inclusion Criteria:

• Age ≥ 18 years
• Patients with acute onset dyspnea (defined as shortness of breath (SOB) at rest or on exertion) and diagnostic uncertainty of etiology where heart failure is in consideration.
• Patients designated to undergo chest X-ray as part of standard of care assessment.
Exclusion Criteria:

• Obvious trauma contributing to dyspnea
• Inability to provide written informed consent
• Not speaking English or Spanish
• Right-sided lobectomy
• Patients with implanted ventricular assist device
• Patient is unable to undergo the TPD test
• Patient is already enrolled in a clinical study with experimental medications
Device: Lung Doppler Signals
ADHF, Lung/Thoracic
ADHF Dyspnea ED
Parkland Health & Hospital System
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Safety and Efficacy Study of IMSA101 in Refractory Malignancies

Open-label, dose escalation (Phase I) and dose expansion (Phase IIA) study of patients receiving intra-tumoral IMSA101 alone or in combination with an immune checkpoint inhibitor (ICI) (Phase I and II)
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Syed Kazmi
177531
All
18 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04020185
STU-2019-0925
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Inclusion Criteria:
1. Signed informed consent and mental capability to understand the informed consent 2. Male or female patients > 18 years of age 3. Histologically or cytologically documented locally advanced or metastatic solid tumor malignancies refractory to or otherwise ineligible for treatment with standard-of-care agents/regimens, including but not limited to:
• Malignant melanoma
• Hormone receptor negative breast cancer
• Gastro-esophageal cancer
• Non-small cell lung cancer
• Head and neck cancer
• Hepatoma
• Renal cell carcinoma 4. Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 1 5. Evaluable or measurable disease as follows:
• A minimum of 3 RECIST-evaluable lesions: one that is suitable for injection and biopsied; one non-injected that will be biopsied for abscopal effect; and one measurable lesion that will be followed for response only.
• Injectable tumors shall be accessed by intralesional (cutaneous) or percutaneous injection only, including those lesions that are visible, palpable, or detectable by standard radiographic or ultrasound methods. Neither surgical procedures nor endoscopically-guided injections including those to endobronchial, endoluminal, or endosinusial spaces shall be allowed. While no anatomic locations are required or disallowed, lesions selected for intratumoral injection must, in the opinion of the investigator:
• Not be immediately adjacent to blood vasculature or other physiologic landmarks in such a way that will accrue undue safety risk to the patient
• Have longest diameter ≥ 10 mm and ≤ 50 mm
• Be fully efficacy evaluable per RECIST v1.1 criteria 6. Life expectancy > 3 months (Phase I) and > 6 months (Phase IIA) 7. ECG without evidence of clinically meaningful conduction abnormalities or active ischemia as determined by the investigator 8. Acceptable organ and marrow function as defined below:
• Absolute neutrophil count > 1,500 cells/μL
• Platelets > 50,000 cells/μL
• Total bilirubin ≤ 1.5 times the upper limit of normal (ULN)
• Aspartate aminotransferase (AST)/alanine transaminase (ALT) ≤ 2.5 times ULN. If liver metastases are present, AST/ALT < 5 times ULN
• Serum creatinine < 1.5 mg/dL and a measured creatinine clearance ≥ 50 mL/min using the Cockcroft-Gault formula
• Prothrombin time (PT)/partial thromboplastin time (PTT) ≤ 1.5 times ULN 9. Women of child-bearing potential (defined as a female who has experienced menarche and who has not undergone successful surgical sterilization (hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) or is not postmenopausal (defined as amenorrhea for at least 12 consecutive months with an appropriate clinical profile at the appropriate age, e.g., greater than 45 years) must have a negative serum pregnancy test prior to first dose of study drug 10. Male and female patients with reproductive potential must agree to use two forms of highly effective contraception throughout the study 11. Phase I combination only: Demonstrated RECIST stable disease through ≥ 4 consecutive cycles of an approved PD-1 or PD-L1 targeted ICI with no Grade ≥ 3 CTCAE events considered by the investigator to be drug-related.
Exclusion Criteria:
1. Anti-cancer therapy within 4 weeks or < 5 half-lives of the first dose of study drug. 2. Failure to recover to Grade 1 or less from clinically significant AEs due to prior anti-cancer therapy. 3. Known untreated brain metastases or treated brain metastases that have not been stable (scan showing no worsening of central nervous system (CNS) lesion[s] and no requirement of corticosteroids) ≥ 4 weeks prior to study enrollment 4. Baseline prolongation of QT/QTc interval (QTc interval > 470) 5. Uncontrolled intercurrent illness (including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations) that in opinion of the investigator would limit compliance with study requirements 6. Women who are pregnant or breastfeeding 7. Phase I combination only: Prior tumor progression through PD-1 or PD-L1 targeted ICI therapy.
Drug: IMSA101, Drug: Immune checkpoint inhibitor (ICI), Drug: Immuno-oncology (IO) therapy
Solid Tumor, Adult, Anus, Breast - Female, Colon, Esophagus, Kidney, Liver, Lung/Thoracic, Melanoma, skin, Other Skin, Pancreas, Rectum, Urinary Bladder
UT Southwestern
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Durvalumab vs Placebo Following Stereotactic Body Radiation Therapy in Early Stage Non-small Cell Lung Cancer Patients (PACIFIC-4)

This is a Phase III, randomized, placebo-controlled, double-blind, multi-center study assessing the efficacy and safety of durvalumab versus placebo following SoC SBRT in patients with unresected clinical Stage I/II lymph node-negative (T1 to T3N0M0) NSCLC.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Puneeth Iyengar
116037
All
18 Years to 130 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03833154
STU-2019-0858
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Key
Inclusion Criteria:
1. Age ≥18 years 2. Histologically or cytologically documented Stage I to II NSCLC, with clinical Stage I/II lymph node-negative (T1 to T3N0M0) disease and planned to receive definitive treatment with SBRT. Patients may be medically inoperable or are medically operable and refusing surgery or choosing to have SBRT (Stereotactic Body Radiation Therapy) as definitive therapy 3. Completion of SoC SBRT as definitive treatment prior to randomization 4. World Health Organization (WHO)/Eastern Cooperative Oncology Group (ECOG) PS of 0, 1, or 2 5. Life expectancy of at least 12 weeks 6. Body weight >30 kg 7. Tumor sample required 8. Adequate organ and marrow function required 9. Patients with central or peripheral lesions are eligible 10. Staging studies must be done within 8 weeks before randomization Key
Exclusion Criteria:
1. Mixed small cell and non-small cell cancer histology 2. History of allogeneic organ transplantation 3. History of another primary malignancy with exceptions 4. History of active primary immunodeficiency 5. Any unresolved toxicity National Cancer Institute (NCI) CTCAE Grade ≥2 from SBRT (Stereotactic Body Radiation Therapy)
Drug: Durvalumab, Other: Placebo
Carcinoma, Non-Small-Cell Lung, Lung/Thoracic
NSCLC, Double- Blind, PD-L1, MEDI4736, Durvalumab, PFS, OS
UT Southwestern; Parkland Health & Hospital System
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The Budesonide in Babies (BiB) Trial (BiB)

This is a Phase 3, randomized, masked, active-controlled, multicenter trial designed to determine whether early intratracheal administration of a combination of budesonide with surfactant, as compared to surfactant alone, will reduce the incidence of physiologic bronchopulmonary dysplasia (BPD) or death by 36 weeks' post-menstrual age in extremely preterm infants.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Joanne.Duran@UTSouthwestern.edu
Luc Brion
85893
All
up to 48 Hours old
Phase 3
This study is NOT accepting healthy volunteers
NCT04545866
STU-2020-0878
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Inclusion Criteria:

• Liveborn infants 22 0/7
•28 6/7 weeks gestation or 401
•1000 grams (inclusive) birth weight
• Clinical decision to give surfactant
• Less than or equal to 48 hours postnatal age
Exclusion Criteria:

• Terminal illness (heart rate < 100 beats per minute, unresponsiveness to resuscitation) or unlikely to survive as judged by the clinician
• Decision to redirect or limit support
• Use of surfactant before enrollment (first dose of surfactant must be study drug)
• Infant received systemic steroids prior to enrollment
• Use of indomethacin, either received by the mother within 24 hours prior to delivery,received by the infant prior to enrollment, or intent to administer to the infant for IVH prophylaxis or PDA management from enrollment up to 7 days of final dose of study drug
• Serious chromosomal abnormalities or major malformations
• Known congenital infections including, but not limited to, confirmed sepsis, congenital CMV, etc.
• Infants with a permanent neuromuscular condition that affects respiration
• Enrollment in a conflicting clinical trial
Drug: budesonide (Pulmicort nebulizing suspension)., Drug: surfactant (poractant alfa,Curosurf)
Respiratory Distress Syndrome, Bronchopulmonary Dysplasia (BPD), Neonatal, Lung/Thoracic, Prematurity, Extreme
UT Southwestern; Children’s Health; Parkland Health & Hospital System
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Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator Therapy (SIMPLIFY)

Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating CF, it is still largely unknown whether or not other chronic therapies can be safely stopped. The SIMPLIFY study is being done to test whether or not it is safe to stop taking inhaled hypertonic saline or Pulmozyme® (dornase alfa) in those people that are also taking Trikafta™. Trikafta (elexacaftor/tezacaftor/ivacaftor) is a combination CFTR modulator therapy that was approved by the Food and Drug Administration for people with CF who have at least one F508del mutation. The three drugs that make up Trikafta work together to allow many more chloride ions to move into and out of the cells, improving the balance of salt and water in the lungs. These changes result in better clearance of mucus from the lungs and improvements in lung function. Inhaled hypertonic saline and Pulmozyme (dornase alfa) also improve clearance of mucus from the lungs to support lung function and have been available to people with CF for many years. Both therapies are considered to be relatively burdensome and it is not known whether either therapy can improve or maintain lung function above what is already gained through Trikafta use. The goal of the SIMPLIFY study is to get information about whether or not it is safe to stop either inhaled hypertonic saline or Pulmozyme (dornase alfa) by testing if there is a change in lung function in subjects with cystic fibrosis (CF) who are assigned to stop their chronic medication (either hypertonic saline or Pulmozyme) as compared to those who are assigned to keep taking their medication while continuing to take Trikafta.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Maria.Mcleod@UTSouthwestern.edu
Raksha Jain
19733
All
12 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT04378153
STU-2020-0246
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Inclusion Criteria:

• Diagnosis of CF.
• Age ≥ 12 years at the Screening Visit.
• Forced expiratory volume in 1 second (FEV1) ≥ 70 % predicted at the Screening Visit if < 18 years old, and ≥ 60 % predicted at Screening Visit if ≥ 18 years old.
• Clinically stable with no significant changes in health status within the 7 days prior to and including the Screening Visit.
• Current treatment with elexacaftor/tezacaftor/ivacaftor (ETI) for at least the 90 days prior to and including the Screening Visit and willing to continue daily use for the duration of the study.
• Currently taking hypertonic saline (at least 3%) and/or dornase alfa for at least the 90 days prior to and including the Screening Visit and willing to continue daily use for the 2-week screening period.
Exclusion Criteria:

• Active smoking or vaping.
• Use of an investigational drug within 28 days prior to and including the Screening Visit.
• Changes to chronic therapy (e.g., ibuprofen, azithromycin, inhaled tobramycin, aztreonam lysine) within 28 days prior to and including the Screening Visit. This includes new airway clearance routines.
• Acute use of antibiotics (oral, inhaled or IV) or acute use of systemic corticosteroids for respiratory tract symptoms within 7 days prior to and including the Screening Visit.
• Chronic use of systemic corticosteroids at a dose equivalent to ≥ 10mg per day of prednisone within 28 days prior to and including the Screening Visit.
• Antibiotic treatment for nontuberculous mycobacteria (NTM) within 28 days prior to and including the Screening Visit.
Other: Discontinuation of hypertonic saline (HS), Other: Continuation of hypertonic saline (HS), Other: Discontinuation of dornase alfa (dnase), Other: Continuation of dornase alfa (dnase)
Cystic Fibrosis
Cystic Fibrosis, CF, Withdrawal, Trikafta, hypertonic saline, dornase alfa, pulmozyme
UT Southwestern
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Evaluation of Efficacy and Safety of Pamrevlumab in Patients With Idiopathic Pulmonary Fibrosis

This is a Phase 3 trial to evaluate the efficacy and safety of 30 mg/kg intravenous (IV) infusions of pamrevlumab administered every 3 weeks as compared to placebo in subjects with Idiopathic Pulmonary Fibrosis
Call 214-648-5005
studyfinder@utsouthwestern.edu, Taelor.Dumond@UTSouthwestern.edu
John Fitzgerald
12247
All
40 Years to 85 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03955146
STU-2019-0797
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Key
Inclusion Criteria:
1. Diagnosis of IPF as defined by ATS/ERS/JRS/ALAT guidelines (Raghu 2018) within the past 7 years prior to study participation. 2. HRCT scan at Screening, with ≥10% to <50% parenchymal fibrosis (reticulation) and <25% honeycombing. 3. FVCpp value >45% and <95% 4. Diffusing capacity of the lungs for carbon monoxide (DLCO) percent predicted ≥25% and ≤90% at screening (determined locally). 5. Not currently receiving treatment for IPF with an approved therapy (i.e., pirfenidone or nintedanib) for any reason, including prior intolerance to an approved IPF therapy. Key
Exclusion Criteria:
1. Previous exposure to pamrevlumab. 2. Evidence of significant obstructive lung disease. 3. Female subjects who are pregnant or nursing. 4. Smoking within 3 months of Screening and/or unwilling to avoid smoking throughout the study. 5. Interstitial lung disease other than IPF. 6. Sustained improvement in the severity of IPF. 7. Other types of respiratory diseases including diseases of the airways, lung parenchyma, pleural space, mediastinum, diaphragm, or chest wall. 8. Certain medical conditions, including recent (e.g. MI/stroke, or severe chronic heart failure or pulmonary hypertension, or cancers. 9. Acute IPF exacerbation during Screening or Randomization. 10. Recent use of any investigational drugs or unapproved therapies, or approved or participation in any clinical trial. 11. History of allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies.
Drug: Pamrevlumab, Drug: Placebo
Idiopathic Pulmonary Fibrosis, Lung/Thoracic
Idiopathic Pulmonary Fibrosis, IPF, Idiopathic Interstitial Pneumonia, Interstitial Lung Disease, Lung Fibrosis
UT Southwestern
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Phase I/II Study of SBRT and GC4711 for Centrally Located or Large NSCLC (GRECO-1)

GTI-4711-101 is a Phase I/II study of the safety of GC4711, its effect on in-field tumor response and its potential to reduce radiation-related pulmonary injury due to SBRT for lymph node negative (T1 to T3N0M0) peripheral or central localized (within 2cm of the proximal bronchial tree) NSCLC. After an open-label, Phase 1, safety cohort of 5 subjects has been completed, a randomized, placebo-controlled Phase 2 portion of 66 subjects will be conducted.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Puneeth Iyengar
116037
All
18 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04476797
STU-2019-1764
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Inclusion Criteria:
1. Male or female subjects at least 18 years of age. 2. Ability to understand and the willingness to sign a written informed consent. 3. Histological or biopsy proven NSCLC. 4. ECOG performance status of 0-3. 5. Node negative (T1 to T3N0M0), centrally located (within 2cm in all directions around the proximal bronchial tree, including ultra-central tumors, abutting the bronchial tree or trachea) or large (>1-7cm) Non-Small Cell Lung Cancer (NSCLC), judged acceptable for SBRT by the treating Investigator 6. Adequate end-organ function, based on routine clinical and laboratory workup: 1. ANC >1,000 cells/µl, Platelets ≥ 75,000 cells/µl, Hemoglobin ≥ 7.0 g/dl 2. Serum creatinine ≤ 2 x ULN or calculated creatinine clearance ≥ 30 ml/min 3. Total bilirubin ≤ 1.5 x ULN (or direct bilirubin below the ULN), AST and ALT ≤ 2.5 x ULN 7. Males and females of must agree to use effective contraception starting prior to the first day of treatment and continuing after the last dose of GC4711/Placebo for 30 days (females) and 90 days (males).
Exclusion Criteria:
1. Subjects with confirmed nodal and/or distant disease(including brain), according standard workup by local investigator 2. Subjects with peripheral lesions 1cm or smaller 3. Prior treatment with immunotherapy within 3 months prior to Day 1 dosing. 4. Prior intra-thoracic radiotherapy or surgery with substantial overlap to planned radiation fields as determined by the treating radiation oncologist. 5. Subjects not recovered/controlled from prior treatment-related (chemotherapy or targeted therapy) toxicities judged by treating physician. 6. Uncontrolled malignancy other than lung cancer that requires active treatment or is deemed by the treating physicians to be likely to affect the subject's survival duration. 7. History of allergic reactions attributed to compounds of similar chemical or biologic composition to GC4711. 8. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that, in the opinion of the investigator, would limit compliance with study requirements. 9. Participation in other clinical trials actively testing new anti-cancer treatments, unless previously written approval is provided by the Sponsor. 10. Requirement for concurrent treatment with nitrates or other drugs that may, in the judgment of the treating investigator, create a risk for a precipitous decrease in blood pressure. 11. Female subjects who are pregnant or breastfeeding. 12. Any other conditions that, in the Investigator's opinion, might indicate the subject to be unsuitable for the study.
Drug: GC4711, Drug: Placebo
NSCLC, Lung/Thoracic, SBRT, Non-metastatic
lymph node negative NSCLC, centrally located NSCLC, large NSCLC, SBRT
UT Southwestern; Parkland Health & Hospital System
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Phase II Randomized Trial of Carboplatin+Pemetrexed+Bevacizumab+/- Atezolizumab in Stage IV NSCLC

While cigarette smoking remains the primary cause of most lung cancer cases, lung carcinoma in never smokers account for nearly 20 percent of cases. Never smokers with lung cancer typically present with different molecular profiles from that of smokers, which results in prognostic and therapeutic implications. Molecular changes in NSCLC that have therapeutic significance include mutations in the epidermal growth factor receptor (EGFR) and rearrangements in the anaplastic lymphoma kinase (ALK) gene. These driver mutations typically are present in lung tumors found in never or light smokers. The addition of bevacizumab to carboplatin and paclitaxel in first-line treatment of non-squamous NSCLC showed improved survival compared to carboplatin and paclitaxel alone, 12.3 vs. 10.3 months respectively. Results from the POINTBREAK trial demonstrated that carboplatin + pemetrexed + bevacizumab is an alternative option to carboplatin + paclitaxel + bevacizumab, with comparable survival but less toxicity. In recent years, immunotherapy has emerged as a form of treatment that can lead to robust responses in a subset of patients. The PD-1 inhibitor nivolumab and the PD-L1 inhibitor atezolizumab have shown prolonged survival in comparison to docetaxel in patients who previously progressed with chemotherapy, irrespective of PD-L1 expression. Thus, this study combines immunotherapeutic agent atezolozumab with an ant-angiogenic agent, bevacizumab, and double platinum therapy (carboplatin and pemetrexed).
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Jonathan Dowell
11902
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT03786692
STU-2020-1121
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Inclusion Criteria:

• Patients must have histologically or cytologically confirmed stage IV non-squamous non-small cell lung cancer
• Patients must either have tumors that harbor an EGFR mutation in exon 19 or exon 21, or must be never smoker wild-types. Never smoker wild-types are defined as patients with tumors without an ALK or ROS1 rearrangement, and are not harboring any EGFR mutation (this includes exons 19 or 21, exon 20, and any other rare EGFR mutations). Never smoker wild-type patients must have smoked less than 100 cigarettes in a lifetime. Patients with an EGFR mutation in exon 19 or 21 may be included irrespective of their smoking history. If tissue-based testing for EGFR mutation status is not available, blood-based EGFR testing that confirms presence of a mutation in exon 19 or 21 is acceptable, and these patients may be included in the study
• Patients must have measurable disease by CT or MRI, defined as at least one lesion that can be accurately measured in at least one dimension in accordance with RECIST criteria v 1.1
• Patients with tumors that harbor an EGFR exon 19 or exon 21 mutation must have received prior treatments with one or more TKIs. A washout period of at least 2 weeks is required to begin treatment in this trial. Patients who are never smoker wild-types must be treatment naïve
• All patients must be chemotherapy, VEGF therapy, and immunotherapy naive, with the exception of prior oral TKIs which are required for EGFR mutated patients. The number of prior oral TKIs and duration of use is neither specified nor limited.
• Patients with a history of treated asymptomatic CNS metastases are eligible, provided they meet all of the following criteria:
• Only supratentorial and cerebellar metastases allowed (i.e., no metastases to midbrain, pons, medulla or spinal cord)
• No ongoing requirement for corticosteroids as therapy for CNS disease
• No stereotactic radiation within 7 days or whole-brain radiation within 14 days prior to randomization
• No evidence of interim progression between the completion of CNS-directed therapy and the screening radiographic study Patients with new asymptomatic CNS metastases detected at the screening scan must receive radiation therapy and/or surgery for CNS metastases. Following treatment, these patients may then be eligible without the need for an additional brain scan prior to randomization, if all other criteria are met
• Age > 18 years
• ECOG performance status 0 or 1
• Patients must have normal organ and marrow function as defined below. The use of G-CSF should follow standard recommendations and physician discretion. If blood transfusion is performed for achieving hemoglobin levels, the levels should stay at ≥ 9.0 mg/ml for at least a week after transfusion. Absolute neutrophil count > 1,500/mcL Hemoglobin ≥ 9.0 mg/ml Platelets > 100,000/mcL Total bilirubin ≤1.5 X institutional upper limit of normal (ULN) AST/ALT (SGOT/SGPT) < 3 times institutional normal limits, or up to 5 times institutional normal limits if the patient has liver metastases Creatinine OR Creatinine clearance ≤1.5 X ULN, OR > 40 Ml/min/1.73 m2 for patients with creatinine levels above institutional normal as per Cockcroft-Gault formula International Normalized Ratio (INR) or Prothrombin Time (PT) ≤1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants Activated Partial Thromboplastin Time (aPTT) <1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants Thyroid stimulating hormone (TSH) Within normal limits a a: If TSH is not within normal limits at baseline, the subject will still be eligible if total T3 or free T4 are within normal limits.
• Patients on full-dose anticoagulation must be on a stable dose (minimum duration 14 days) of oral anticoagulant or low molecular weight heparin (LMWH). If receiving warfarin, the patient must have an INR ≤3.0. For heparin and LMWH there should be no clinically significant active bleeding (with no bleeding within 14 days prior to first dose of protocol therapy) or pathological condition present that carries a high risk of bleeding (for example, tumor involving major vessels or known varices).
• Ability to understand and willingness to sign a written informed consent and HIPAA consent document.
• A core biopsy must be available for the study. The biopsy sample must be adequate for analyses. If the sample is not adequate, the patient must agree to provide a fresh biopsy specimen before the start of treatment. Any available archival tissue will also be collected.
• Urinary protein must be ≤1+ on dipstick or routine urinalysis (UA; if urine dipstick or routine analysis is ≥2+, a 24 hour urine collection for protein must demonstrate <1000 mg of protein in 24 hours to allow participation in the protocol).
• Female subjects of child-bearing potential must be willing to use an effective method of contraception, for the course of the study through at least 6 months after the last dose of study medication.
• Male patients who have WOCBP partners must agree to use effective method of contraception for the course of the study through 8 months after the last dose of study medication.
• Note: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject.
Exclusion Criteria:

• Patients currently receiving any other investigational agents, immunomodulatory agents, chemotherapy, or TKIs. EGFR mutation-positive patients must have received prior TKI treatment
• The patient has experienced any Grade 3-4 GI bleeding within 3 months prior to first dose of protocol therapy.
• The patient has a history of deep vein thrombosis (DVT), pulmonary embolism (PE), or any other significant thromboembolism (venous port or catheter thrombosis or superficial venous thrombosis are not considered "significant") during the 3 months prior to the first dose of protocol therapy.
• Subjects with untreated CNS metastases are excluded, even if they are asymptomatic. Patients with treated brain metastases will be allowed if brain imaging obtained within 28 days of trial enrollment reveals stable disease.
• Cirrhosis at a level of Child-Pugh B or worse, or cirrhosis of any degree and a history of hepatic encephalopathy, or clinically meaningful ascites resulting from cirrhosis. Clinically meaningful ascites is defined as ascites from cirrhosis requiring diuretics or paracentesis.
• The patient has experienced any arterial thromboembolic events, including but not limited to myocardial infarction, transient ischemic attack, cerebrovascular accident, or unstable angina, within 6 months prior to first dose of protocol therapy.
• The patient has uncontrolled or poorly-controlled hypertension (>150 mmHg systolic or > 100 mmHg diastolic for >4 weeks) despite standard medical management
• Prior history of hypertensive crisis or hypertensive encephalopathy
• Significant vascular disease (e.g., aortic aneurysm requiring surgical repair or recent peripheral arterial thrombosis) within 6 months prior to randomization
• Evidence of bleeding diathesis or coagulopathy (in the absence of therapeutic anticoagulation)
• History of abdominal or tracheosphageal fistula or gastrointestinal perforation within 6 months prior to randomization
• Clinical signs of gastrointestinal obstruction or requirement for routine parenteral hydration, parenteral nutrition, or tube feeding
• Evidence of abdominal free air not explained by paracentesis or recent surgical procedure
• Serious, non-healing wound, active ulcer, or untreated bone fracture within 28 days prior to first dose of protocol therapy
• Subjects with a history of smoking greater than a 100 cigarettes in a lifetime, unless their tumor has an EGFR exon 19 or exon 21 mutation.
• Patients with active, suspected, or known autoimmune disease that has required systemic treatment in the past one year (i.e., with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Hormone replacement therapy (e.g. thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
• Patients with a history of hemoptysis (defined as bright red blood or ≥1/2 teaspoons) within 1 month prior to first dose of protocol therapy or with radiographic evidence of major blood vessel invasion or encasement by cancer.
• The patient has undergone major surgery within 28 days prior to first dose of study treatment, or minor surgery/ subcutaneous venous access device placement within 7 days prior to first dose of protocol therapy. The patient has elective or planned major surgery to be performed during the course of the clinical trial.
• The patient is receiving chronic anti-platelet therapy other than aspirin, including non-steroidal anti-inflammatory drugs (NSAIDs, including ibuprofen, naproxen, and others), dipyridamole or clopidogrel, or similar agents. Once-daily aspirin use (maximum dose 325 mg/day) is permitted. Occasional use of NSAIDs is allowed (for example daily use for less than a week; treating physician discretion is permitted to differentiate between occasional vs chronic use)
• Patients who have not recovered from adverse events due to agents administered earlier except neuropathy and alopecia. Physician's discretion is allowed to decide which unresolved adverse events from previous therapy (for NSCLC) prohibit patient participation in this study.
• Patients requiring more than 10 mg prednisolone (or its equivalent) per day are excluded.
• Patients with any evidence of interstitial lung disease (ILD) or pneumonitis or a prior history of ILD or pneumonitis requiring oral or IV glucocorticoids. History of radiation pneumonitis in the radiation field (fibrosis) is permitted
• Patients with active tuberculosis infection are excluded.
• Patients who have received a live vaccine within 30 days prior to cycle 1 Day 1.
• Uncontrolled illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia (significant), cirrhosis, or psychiatric illness/ social situations that would limit compliance with the study requirements.
• Known history of testing positive for immunodeficiency virus (HIV) or known acquired immunodeficiency syndrome (AIDS).
• Known history of chronic hepatitis B virus infection or chronic hepatitis C virus indicating chronic infection that is not cured.
• Subjects with previous malignancies (except non-melanoma skin cancers, and in situ cancers, such as, bladder, gastric, colon, cervical/ dysplasia, melanoma, or breast) are excluded unless a complete remission was achieved at least 2 years prior to study registration and no additional therapy is required or anticipated to be required during the study period.
• Leptomeningeal disease
• Uncontrolled tumor-related pain Patients requiring pain medication must be on a stable regimen at study entry. Symptomatic lesions amenable to palliative radiotherapy (e.g., bone metastases or metastases causing nerve impingement) should be treated prior to randomization. Patients should be recovered from the effects of radiation. There is no required minimum recovery period. Asymptomatic metastatic lesions whose further growth would likely cause functional deficits or intractable pain (e.g., epidural metastasis that is not currently associated with spinal cord compression) should be considered for locoregional therapy, if appropriate, prior to randomization.
• Uncontrolled pleural effusion, pericardial effusion, or ascites requiring recurrent drainage procedures (once monthly or more frequently). Patients with indwelling catheters (e.g., PleurX®) are allowed.
• Ca > 12 mg/dl or corrected serum calcium > ULN Patients who are receiving denosumab prior to randomization must be willing and eligible to receive a bisphosphonate instead while in the study
• Pregnant or breast feeding
• Prior allogeneic bone marrow transplantation or solid organ transplant
• Known hypersensitivity to Chinese hamster ovary cell products or any of the study drugs.
• Clear tumor infiltration into the thoracic great vessels is seen on imaging
• Clear cavitation of pulmonary lesions is seen on imaging
• Subjects with squamous cell carcinoma of the lung.
• Subjects with a lung tumor with a known ALK or ROS1 rearrangement or an EGFR mutation other than in exon 19 or exon 21.
Drug: Arm A, Drug: Arm B
Lung/Thoracic, Non-Small Cell Carcinoma of Lung, TNM Stage 4
UT Southwestern
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Durvalumab and Stereotactic Radiotherapy for Advanced NSCLC

This is a randomized Phase II study which is designed to determine the impact of stereotactic radiotherapy and durvalumab on quality-of-life and oncologic outcomes in patients with advanced non-small cell lung cancer. Durvalumab (Imfinzi) and stereotactic radiotherapy, with each fraction of radiotherapy is given every other day on a standard stereotactic ablative radiotherapy (SAbR) schedule or every four weeks on the personalized ultra-fractionated stereotactic adaptive radiotherapy (PULSAR) schedule. Subjects will be followed for a period of 2 years after completion of treatment or until death, whichever occurs first. Specifically, subjects will be followed at 1, 3, 6, 9, 12, 15, 18, 21, and 24 months following treatment. After the 2 year follow up, the patient can continue routine follow up with their physicians, per standard of care. Subjects removed from therapy for unacceptable adverse events will be followed until resolution or stabilization of the adverse event.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Dat Vo
164202
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT04786093
STU-2021-0171
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Inclusion Criteria:

• Patients must have biopsy-proven metastatic non-small cell lung cancer and eligible for receipt of immunotherapy Patients can present with either de novo metastatic disease or recurrent disease Patients must have at least one (1) symptomatic or progressive metastatic sites with no more than 10 metastatic sites, based on standard imaging studies Patients cannot have received any prior radiation therapy or surgery to the intended radiation treatment area (index lesion) Patients with brain metastases may be enrolled if all lesions are treated with radiation therapy or surgery prior to start of protocol therapy Metastases in major lower extremity weight-bearing bones or spine should undergo surgical stabilization if indicated Age greater than or equal to 18 years. Both men and women and members of all races and ethnic groups will be included Eastern Cooperative Oncology Group Performance status 0 to 2 (Appendix A) Adequate normal organ and bone marrow function as defined by:
• Haemoglobin ≥9.0 g/dL
• Absolute neutrophil count (ANC) ≥ 1.0 × 109 /L
• Platelet count ≥75 × 109/L
• Serum bilirubin ≤1.5 x institutional upper limit of normal (ULN). This will not apply to patients with confirmed Gilbert's syndrome (persistent or recurrent hyperbilirubinemia that is predominantly unconjugated in the absence of hemolysis or hepatic pathology), who will be allowed only in consultation with their physician.
• AST (SGOT)/ALT (SGPT) ≤2.5X institutional upper limit of normal unless liver metastases are present, in which case it must be ≤5X ULN
• Measured creatinine clearance (CL) >40 mL/min or Calculated creatinine CL>40 mL/min by the Cockcroft-Gault formula (Cockcroft and Gault 1976) or by 24-hour urine collection for determination of creatinine clearance: Males: Creatinine CL (mL/min) = Weight (kg) x (140
•Age) 72 x serum creatinine (mg/dL) Females: Creatinine CL (mL/min) = Weight (kg) x (140
•Age) x 0.85 72 x serum creatinine (mg/dL) All men, as well as women of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 90 days following completion of therapy. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. Medically accepted forms of birth control include male condoms plus spermicide, diaphragm, cervical cap, the placement of a Copper T intrauterine device (IUD), birth control pills, Levonorgesterel-releasing intrauterine system (IUS), hormone implants or injections, or combined pill, minipill patch, or a partner who has undergone a vasectomy (surgical sterility). A female of child-bearing potential is any woman (regardless of sexual orientation, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria:
• Has not undergone a hysterectomy or bilateral oophorectomy; or
• Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months). Life expectancy greater than six (6) months Body weight greater than 30 kg Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Written informed consent and any locally required authorization (e.g., Health Insurance Portability and Accountability Act in the US, European Union [EU] Data Privacy Directive in the EU) obtained from the patient/legal representative prior to performing any protocol-related procedures, including screening evaluations. Patient is willing and able to comply with the protocol for the duration of the study including undergoing treatment and scheduled visits and examinations including follow up.
Exclusion Criteria:

• Active or prior documented autoimmune or inflammatory disorders (including inflammatory bowel disease [e.g., colitis or Crohn's disease], diverticulitis [with the exception of diverticulosis], systemic lupus erythematosus, Sarcoidosis syndrome, or Wegener syndrome [granulomatosis with polyangiitis, Graves' disease, rheumatoid arthritis, hypophysitis, uveitis, etc]). The following are exceptions to this criterion:
• Patients with vitiligo or alopecia
• Patients with hypothyroidism (e.g., following Hashimoto syndrome) stable on hormone replacement
• Any chronic skin condition that does not require systemic therapy
• Patients without active disease in the last 5 years may be included but only after consultation with the study physician
• Patients with celiac disease controlled by diet alone Administration of two or more lines of systemic therapy for the diagnosis of metastatic lung cancer • Prior receipt of systemic therapy for the management of high-risk early stage or locally advanced non-small cell lung cancer, prior to the development of metastatic disease, would not count towards the number of receipt of systemic therapy Subjects may not be receiving any other investigational agents for the treatment of the cancer under study. Patients with untreated brain metastases Patients with progressive metastatic disease involving the skin or subcutaneous tissues, esophagus, stomach, intestines, or mesenteric lymph nodes that are felt to be too high risk to treat with radiation therapy to protocol dose. Patients cannot have pathologic fracture at the evaluated site Patients cannot have untreated spinal cord compression History of allergic reactions attributed to compounds of similar chemical or biologic composition to durvalumab or other agents used in study Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that, in the opinion of the investigator, would limit compliance with study requirements Subjects must not be pregnant or nursing due to the potential for congenital abnormalities and the potential of this regimen to harm nursing infants Male or female patients of reproductive potential who are not willing to employ effective birth control from screening to 90 days after the last dose of durvalumab monotherapy Participation in another clinical study with an investigational product during the last 3 months Concurrent enrolment in another clinical study, unless it is an observational (non-interventional) clinical study or during the follow-up period of an interventional study Receipt of the last dose of anticancer therapy (chemotherapy, immunotherapy, endocrine therapy, targeted therapy, biologic therapy, tumor embolization, monoclonal antibodies) ≤7 days prior to the first dose of study drug If sufficient wash-out time has not occurred due to the schedule or PK properties of an agent, a longer wash-out period will be required, as agreed by AstraZeneca/MedImmune and the investigator Any unresolved toxicity NCI CTCAE Grade ≥2 from previous anticancer therapy with the exception of alopecia, vitiligo, and the laboratory values defined in the inclusion criteria:
• Patients with Grade ≥2 neuropathy will be evaluated on a case-by-case basis after consultation with the Study Physician.
• Patients with irreversible toxicity not reasonably expected to be exacerbated by treatment with durvalumab may be included only after consultation with the Study Physician. Any concurrent chemotherapy, immunotherapy, biologic, or hormonal therapy for cancer treatment. Concurrent use of hormonal therapy for non-cancer-related conditions (e.g., hormone replacement therapy) is acceptable. Major surgical procedure (as defined by the Investigator) within 28 days prior to the first dose of immunotherapy. Note: Local surgery of isolated lesions for palliative intent is acceptable History of allogenic organ transplantation History of another primary malignancy except for:
• Malignancy treated with curative intent and with no known active disease ≥5 years before the first dose of immunotherapy and of low potential risk for recurrence
• Adequately treated non-melanoma skin cancer or lentigo malignant without evidence of disease
• Adequately treated carcinoma in situ without evidence of disease History of leptomeningeal carcinomatosis History of active primary immunodeficiency Active infection including tuberculosis (clinical evaluation that includes clinical history, physical examination and radiographic findings, and TB testing in line with local practice), hepatitis B (known positive HBV surface antigen (HBsAg) result), hepatitis C. Patients with a past or resolved HBV infection (defined as the presence of hepatitis B core antibody [anti-HBc] and absence of HBsAg) are eligible. Patients positive for hepatitis C (HCV) antibody are eligible only if polymerase chain reaction is negative for HCV RNA Current or prior use of immunosuppressive medication within 14 days before the first dose of durvalumab. The following are exceptions to this criterion:
• Intranasal, inhaled, topical steroids, or local steroid injections (e.g., intra articular injection)
• Systemic corticosteroids at physiologic doses not to exceed 10 mg/day of prednisone or its equivalent
• Steroids as premedication for hypersensitivity reactions (e.g., CT scan premedication) Receipt of live attenuated vaccine within 30 days prior to the first dose of immunotherapy. Other forms of vaccines, such as mRNA, recombinant protein, and non-replicating vector-based vaccines, are permitted. Note: Patients, if enrolled, should not receive live vaccine whilst receiving immunotherapy and up to 30 days after the last dose of immunotherapy Receipt of any medications listed below: Patients on this study should not be on any targeted systemic therapies such as those directed at EGFR mutations, ALK or ROS1 gene rearrangements, BRAF V600E mutation, or NTRK gene fusions. Other anti-cancer treatments are also not allowed on the study and are listed below. Supportive medications may be given at any point during treatment at the discretion of the treating physician, such as anti-emetics, pain medications, anti-diarrheals, nutritional supplementations, and anti-depressants. Anti-oxidant medications in excess of daily recommended values are not allowed.
• Any investigational anticancer therapy other than those under investigation in this study should not be given concomitantly whilst the patient is on study treatment.
• mAbs against CTLA-4, PD-1, or PD-L1 other than those under investigation in this study should not be given concomitantly whilst the patient is on study treatment.
• Any concurrent chemotherapy, radiotherapy, immunotherapy, biologic, or hormonal therapy for cancer treatment other than those under investigation in this study should not be given concomitantly whilst the patient is on study treatment. (Concurrent use of hormones for non-cancer-related conditions [e.g., insulin for diabetes and hormone replacement therapy] is acceptable. Local treatment of isolated lesions, excluding target lesions, for palliative intent is acceptable [e.g., by local surgery or radiotherapy])
• Immunosuppressive medications including, but not limited to, systemic corticosteroids at doses exceeding 10 mg/day of prednisone or equivalent, methotrexate, azathioprine, and tumor necrosis factor-α blockers should not be given concomitantly, or used for premedication prior to the IO infusions. The following are allowed exceptions:
• Use of immunosuppressive medications for the management of IP-related AEs,
• Use in patients with contrast allergies.
• In addition, use of inhaled, topical, and intranasal corticosteroids is permitted. A temporary period of steroids will be allowed if clinically indicated and considered to be essential for the management of non-immunotherapy related events experienced by the patient (e.g., chronic obstructive pulmonary disease, radiation, nausea, etc.).
• EGFR tyrosine kinase inhibitors (TKI) should not be given concomitantly, and should be used with caution in the 90 days post last dose of durvalumab.
• Live attenuated vaccines should not be given through 30 days after the last dose of IP (including SoC)
• Herbal and natural remedies which may have immune-modulating effects should not be given concomitantly unless agreed by the sponsor
Radiation: Stereotactic radiation therapy, Drug: Durvalumab
Non Small Cell Lung Cancer, Lung/Thoracic
Non-small cell lung cancer, Immunotherapy, Durvalumab, Radiation therapy, Radiotherapy, Stereotactic body radiation therapy, Stereotactic ablative radiotherapy, SBRT, SABR, PULSAR
UT Southwestern; Parkland Health & Hospital System
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Testicular Tissue Cryopreservation for Fertility Preservation

Testicular tissue cryopreservation is an experimental procedure where a young boy's testicular tissue is retrieved and frozen. This technique is reserved for young male patients who are not yet producing mature sperm, with the ultimate goal that their tissue may be used in the future to restore fertility when experimental techniques emerge from the research pipeline.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Ksenya Shliakhtsitsava
181933
Male
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT02972801
STU-2020-1412
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Inclusion Criteria:

• Be male at any age.
• Be scheduled to undergo surgery, chemotherapy, drug treatment and/or radiation for the treatment or prevention of a medical condition or malignancy with risk of causing permanent and complete loss of subsequent testicular function.
• Or, have a medical condition or malignancy that requires removal of all or part of one or both testicles.
• Have newly diagnosed or recurrent disease. Those who were not enrolled at the time of initial diagnosis (i.e., patients with recurrent disease) are eligible if they have not previously received therapy that is viewed as likely to result in complete and permanent loss of testicular function.
• Have two testicles if undergoing elective removal of all or part of a testicle for fertility preservation only. Note: removal of both testicles will limit fertility preservation options.
• Sign an approved informed consent and authorization permitting the release of personal health information. The patient and/or the patient's legally authorized guardian must acknowledge in writing that consent for specimen collection has been obtained, in accordance with institutional policies approved by the U.S. Department of Health and
• Consent for serum screening tests for infectious diseases [HIV-1, HIV-2, Hepatitis B, Hepatitis C], to be performed at the time of testicular tissue harvesting.
• Undergo a full history and physical examination and obtain standard pre-operative clearance (based on the most recent ACC/AHA Guideline for Perioperative Cardiovascular Evaluation for Noncardiac Surgery) as determined by their primary surgeon.
• Participating in long term follow-up is a requirement of the protocol.
Exclusion Criteria:

• Diagnosed with psychological, psychiatric, or other conditions which prevent giving fully informed consent.
• Diagnosed with an underlying medical condition that significantly increases their risk of complications from anesthesia and surgery.
Procedure: Testicular biopsy
Lymphoma, Sarcoma, Cancer, Mycosis Fungoides, Brain and Nervous System, Other, Eye and Orbit, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Male, Carcinoid Tumor, Cardiovascular, Colon, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Nose, Other Digestive Organ, Other Endocrine System, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Leukemia, Other, Hodgkins Lymphoma, Heart, Leukemia, Not Otherwise Specified, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Non-Hodgkins Lymphoma, Other Hematopoietic, Small Intestine, Soft Tissue, Unknown Sites, Ill - Defined Sites, Autoimmune Disorders
Spermatogonial stem cells, Testis, Fertility, Infertility, Oncofertility
Children’s Health
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Comparing Virtual Reality (VR) to Non-VR for Decreasing Preoperative/Procedural Anxiety

The primary objective of the study is to utilize the modified Yale Preoperative Anxiety scale (mYPAS), a validated preoperative/procedural anxiety score, to measure preoperative anxiety via distraction in pediatric oncology patients undergoing port access. The hypothesis is that using Virtual Reality (VR) will objectively decrease anxiety scores measured by mYPAS by five percent (primary outcome). The secondary outcome will be the parents or the legally authorized representative (LAR) subjective reports of anxiety with the use of VR. The Kind VR device is used in house at Children's Health in the Dallas and Plano campuses. The VR device used in this study qualifies as exempt from FDA IDE regulations. It is a non-significant risk, non-invasive, interactive video device the user wears like goggles. The study carries minimal risks to the subjects and is designed to minimize patient discomfort from placement or motion sickness. Furthermore, the device has disposable covers for protection against infection and can be sanitized between uses, once the disposable covers are removed. Children's Health System of Texas (CHST) and this research group are not partnering entities with the Kind VR, and the Kind VR device is not being studied. The effect of virtual reality (VR) on preprocedural anxiety as measured by questionnaires and the observations of the modified Yale Preoperative Anxiety Scale (mYPAS) is being studied Most patients coming to the Clinic of Cancer and Blood Disorders (CCBD) are under chronic care for their ongoing disease and are likely to be coming to the CCBD at least twice in a 6-month period. The CCBD schedule will be reviewed by the researchers for patients age 5-12, requiring port access at least twice during the next six-month period. Patient families whose child meets the basic screening criteria, and have no exclusion criteria, will be approached privately as possible participants in the study. Up to 100 subjects will be enrolled over a 2-year period. Once the subject/parent or LAR agrees to participate, study staff will randomize the subjects into which standard of care distraction method for anxiety management they will receive first in this study.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Sarah Rebstock
189091
All
5 Years to 12 Years old
N/A
This study is also accepting healthy volunteers
NCT05094141
STU-2020-0607
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Inclusion Criteria:

• Any patient of the Children's Medical Center CCBD
• 5-12 years of age
• Patient requiring their port accessed twice or more within a 6 months period
• Ability to understand and the willingness to sign a written informed consent. Exclusion Criteria
• Subjects younger than 5 and older than 12
• Patients requiring recovery in PICU or sites other than PACU
• If parents or subject is not willing to participate
• Subjects with severe developmental delays and subjects with developmental challenges preventing them from keeping the VR device on are also excluded
• Patients who will not be in CCBD for port access at least twice in 6 months
Other: Virtual reality
Lymphoma, Sarcoma, Multiple Myeloma, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Carcinoid Tumor, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Leukemia, Other, Hodgkins Lymphoma, Leukemia, Not Otherwise Specified, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Non-Hodgkins Lymphoma, Small Intestine, Soft Tissue, Preoperative Anxiety
virtual reality, preoperative anxiety, modified Yale Preoperative Anxiety Scale
Children’s Health
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Non-Invasive Diagnosis of Pediatric Pulmonary Invasive Mold Infections (DOMINIC)

This study will establish a non-invasive diagnostic approach and evaluate clinical outcomes for children at high-risk for pulmonary invasive mold infection (PIMI).
Call 214-648-5005
studyfinder@utsouthwestern.edu, Cameron.James@UTSouthwestern.edu
Paul Sue
157043
All
120 Days to 21 Years old
This study is NOT accepting healthy volunteers
NCT03827694
STU-2019-1188
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Inclusion Criteria:

• Males or females age > 120 days and < 22 years at any participating site
• Have at least one of the following conditions associated with a known high incidence of IMI: hematopoietic stem cell transplantation (HSCT), aplastic anemia, or hematologic malignancy
• New (last 96 hours) radiographic evidence of at least one of the following: at least one nodular lesion greater than or equal to 5 mm in size, a cavitary lesion, a lesion with a halo sign, a lesion with a reverse halo sign, or a lesion with an air crescent sign
• Prolonged neutropenia (absolute neutrophil count < 500 cells/µl for a period of ≥ 5 consecutive days) in 30 days prior to qualifying chest MRI or CT scan date OR currently receiving systemic therapy for acute or chronic graft-versus-host disease (GVHD) on the date of the qualifying chest MRI or CT scan
• Subject consent or parental/guardian permission (informed consent) and if appropriate, child assent
Exclusion Criteria:

• Weight <3 kg, so as to not exceed 3 ml/kg in a single blood draw
• Previous inclusion in this study
Diagnostic Test: Non-Invasive Testing for PIMI
Lung/Thoracic, Pulmonary Invasive Mold Infections, Pulmonary Invasive Aspergillosis
Children’s Health
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IV Gallium Study for Patients With Cystic Fibrosis Who Have NTM (ABATE Study) (ABATE)

The purpose of this study is to assess the safety and tolerability of two 5-day infusion cycles of IV gallium in adult patients with CF who are infected with NTM. Funding Source - FDA OOPD
Call 214-648-5005
studyfinder@utsouthwestern.edu, YAMEI.CHENG@UTSouthwestern.edu
Raksha Jain
19733
All
18 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT04294043
STU-2021-0279
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Inclusion Criteria:
1. Written informed consent obtained from subject or subject's legal representative 2. Be willing and able to adhere to the study visit schedule and other protocol requirements 3. Greater than or equal to 18 years of age at Visit 1 4. Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
• Sweat chloride ≥ 60 milliequivalent (mEq)/liter by quantitative pilocarpine iontophoresis test (QPIT)
• Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
• Abnormal nasal potential difference (NPD) (change in NPD in response to a low chloride solution and isoproteronol of less than -5 mV) 5. Documentation of NTM culture positive defined as follows:
• Two positive NTM culture results from sputum (or BAL) at least 28 days apart (these are the two qualifying positive cultures)
• Both qualifying positive culture results include M. avium complex, M. abscessus complex, or both M. avium and M. abscessus
• Both qualifying positive culture results include the same species or subspecies
• No cultures negative for NTM since the first of the two qualifying positive culture results 6. Current NTM species or subspecies has never been treated or previous treatment was associated with clearance of NTM and completed > 2 years prior to Day 1 7. Forced expiratory volume in 1 second (FEV1) ≥ 25 % of predicted value at Screening 8. Able to expectorate sputum 9. Clinically stable with no significant changes in health status within 7 days prior to Day 1 10. Enrolled in the CFF Cystic Fibrosis Foundation Patient Registry (CFFPR) 11. Willing to discontinue chronic azithromycin use for the duration of the study
Exclusion Criteria:
1. Any of the following abnormal lab values at screening:
• Hemoglobin <10g/dL
• Platelets <100,000/mm3
• White blood cells (WBC) < 4,500/mm3
• Aspartate transaminase (AST), alanine transaminase (ALT), gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) ≥3 x upper limit of normal
• Serum creatinine > 2.0 mg/dl and ≥1.5 x upper limit of normal
• Ionized calcium ≤ lower limit of normal (only performed if total calcium is ≤ lower limit of normal) 2. History of solid organ or hematological transplantation 3. Use of bisphosphonates within 7 days prior to Day 1 4. Known sensitivity to gallium 5. Use of any investigational drug and/or participated in any interventional clinical trial within 28 days prior to Day 1 6. In the opinion of the Investigator, features of active NTM disease are present (e.g., clinical worsening is likely due to NTM disease despite definitive treatment of co-pathogens and/or acute exacerbations) 7. Undergoing treatment for NTM disease or anticipate beginning treatment within 3 months 8. Current diagnosis of osteoporosis 9. For people of childbearing potential:
• Positive pregnancy test at Visit 1 or
• Lactating or
• Unwilling to practice a medically acceptable form of contraception (acceptable forms of contraception: abstinence, hormonal birth control, intrauterine device, or barrier method plus a spermicidal agent), unless surgically sterilized or postmenopausal during the study 10. For people able to father a child: unwilling to use adequate contraception (as determined by the investigator) during the study 11. Has any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives 12. New initiation of chronic therapy (greater than 21 days) within 28 days prior to the Enrollment Visit
Drug: Gallium nitrate
Lung/Thoracic, Nontuberculous Mycobacterium Infection
Cystic Fibrosis, Gallium Nitrate, IV Gallium, Nontuberculous mycobacterium, NTM, Mycobacterium abscessus, mycobacterium avium
UT Southwestern
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Evaluation of Point-of-Care (EPOC) for COVID-19 ((EPOC))

EPOC is designed to examine the feasibility of conducting two point-of-care (POC) tests the LumiraDx SARS-CoV-2 Antibody Test and the RightSign COVID-19 IgG/IgM Rapid Test for SARS-CoV-2 antibodies and compare the reproducibility of these tests to tests run at a central laboratory on specimens obtained from the same study participants at the same time.
Call 214-648-5005
studyfinder@utsouthwestern.edu, TIANNA.PETERSEN@UTSouthwestern.edu
Mamta Jain
41138
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT05227404
STU-2022-0468
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Inclusion Criteria:

• Age ≥ 18 years.
• Informed consent by the patient or the patient's legally authorized representative (LAR) for up to 4 fingersticks for POC testing and a blood draw for stored blood samples.
• SARS-CoV-2 infection, documented by a nucleic acid test (NAT) or equivalent testing within 3 days prior to consent OR documented by NAT or equivalent testing more than 3 days prior to consent AND progressive disease suggestive of ongoing SARS-CoV-2 infection per the responsible investigator. (For non-NAT tests, only those deemed with equivalent specificity to NAT by the protocol team will be allowed. A central list of allowed non-NAT tests is maintained for TICO and that list will also be used for this protocol.)
• Duration of symptoms attributable to COVID-19 ≤ 12 days per the responsible investigator.
• Requiring admission for inpatient hospital acute medical care for clinical manifestations of COVID-19, per the responsible investigator, and NOT for purely public health or quarantine purposes.
Exclusion Criteria:

• Prior receipt of SARS-CoV-2 hIVIG, convalescent plasma from a person who recovered from COVID-19, or SARS-CoV-2 nMAb within 6 months of the blood draws for testing as part of this protocol.
• Disease severity beyond that of stratum 1 in the TICO trial. This includes the following conditions: 1. stroke 2. meningitis 3. encephalitis 4. myelitis 5. myocardial infarction 6. myocarditis 7. pericarditis 8. symptomatic congestive heart failure (CHF; New York Heart Association [NYHA] class III-IV) 9. arterial or deep venous thrombosis or pulmonary embolism
• Current requirement for any of the following: 1. high-flow supplemental oxygen 2. non-invasive ventilation 3. invasive mechanical ventilation 4. extracorporeal membrane oxygenation 5. mechanical circulatory support 6. vasopressor therapy 7. commencement of renal replacement therapy at this admission (i.e., not patients on chronic renal replacement therapy).
• In the opinion of the responsible investigator, any condition for which, participation would not be in the best interest of the participant or that could limit protocol specified assessments.
Diagnostic Test: LumiraDX, Diagnostic Test: RightSign, Diagnostic Test: Case Control
COVID-19, Lung/Thoracic, SARS CoV 2 Infection
COVID-19, SARS COV 2, COVID19 testing
UT Southwestern; Parkland Health & Hospital System
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Prospective Treatment Efficacy in IPF Using Genotype for Nac Selection (PRECISIONS) Trial (PRECISIONS)

The purpose of this study is to compare the effect of n-acetylcysteine (NAC) plus standard care with matched placebo plus standard of care in patients diagnosed with idiopathic pulmonary fibrosis (IPF) who have the TOLLIP rs3750920 TT genotype. The study will compare the time to a composite endpoint of relative decline in lung function [10% relative decline in forced vital capacity (FVC), first respiratory hospitalization, lung transplantation, or all-cause mortality] The secondary objectives will be to examine the effect of NAC on the components of the primary composite endpoint, the rates of clinical events, change in physiology, change in health status, and change in respiratory symptoms.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Nighat.Sultana@UTSouthwestern.edu
Chad Newton
74684
All
40 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04300920
STU-2021-0623
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Inclusion Criteria:

• ≥ 40 years of age
• Diagnosed with IPF according to 2018 ATS/ERS/JRS/ALAT, confirmed by enrolling investigator
• Signed informed consent
• If taking pirfenidone or nintedanib, must be on stable dose for at least 6 weeks prior to enrollment visit
• Confirmed rs3570920 TT TOLLIP genotype
Exclusion Criteria:

• Pregnancy or planning to become pregnant
• Women of childbearing potential not willing to remain abstinent (refrain from heterosexual intercourse) or use two adequate methods of contraception, including at least one method with a failure rate of <1% per year during study participation
• Significant medical, surgical or psychiatric illness that in the opinion of the investigator would affect subject safety, including liver and renal failure
• Receipt of an investigational drug or biological agent within the previous 4 weeks of the screening visit or 5 times the half-life, if longer
• Supplemental or prescribed NAC therapy within 60 days of enrollment
• Listed for lung transplantation at the time of screening
• History of lung cancer
• Inability to perform spirometry
• Forced vital capacity (FVC) less than 45% predicted, using the global lung function index (GLI) equation at Visit 1
• Active respiratory infection requiring treatment with antibiotics within 4 weeks of Visit 1
Drug: N-acetyl cysteine, Drug: Placebo
Idiopathic Pulmonary Fibrosis
IPF, Pulmonary Fibrosis, n-acetylcysteine, NAC
UT Southwestern
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Gender Disparity and Hormones in Cystic Fibrosis

The objective of this study is to investigate the impact of hormones on lung disease in Cystic Fibrosis (CF) patients. Due to improved therapies, CF patients are living longer and healthier lives than they did 20 years ago. However, females have been shown to have a survival disadvantage. The median life expectancy is 33 in women and 37 in men with CF. The hypothesis is that estrogen and/or progesterone negatively impact lung health in CF. Therefore, understanding the impact of sex hormones (including the use of birth control pills) on the disease process is increasingly important. The purpose of this study is to determine if lung function, respiratory symptoms, or various markers of lung health change during different phases of the natural ovulatory cycle in order to understand if estrogen or progesterone hormones are impacting the disease relative to fluctuations in men with stable testosterone levels. The research objectives of this project are to: - Determine if lung function, respiratory symptoms, or various markers of lung health change during different hormonal phases of the ovulatory cycle in women. - Determine if men change lung function, respiratory symptoms, or various markers of lung health over time. - Determine if oral contraceptive pills in women stabilize fluctuations in symptoms and improve lung health.
Call 214-648-5005
studyfinder@utsouthwestern.edu, LYNN.FERNANDEZ@UTSouthwestern.edu
Raksha Jain
19733
All
18 Years and over
Early Phase 1
This study is NOT accepting healthy volunteers
NCT02036879
STU 072013-079
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Inclusion Criteria:

• Male and female cystic fibrosis patients
• Must be greater than or equal to 18 years of age.
• All subjects must understand and sign the informed consent.
• Subjects must have the ability to read and write in English.
• Female subjects starting this study must be willing to use a double barrier method of birth control (such as condom or diaphragm) used with a spermicide (a substance that kills sperm), while participating in the study. Exclusion Criteria for Main Study:
• Women who are pregnant, breast feeding, or who have had an oophorectomy.
• Women who have received a hormone contraceptive injection (such as Depo Provera) within the last 3 months. Exclusion Criteria for Substudy:
• Men
• Women who have a history of breast cancer, abnormal vaginal bleeding, liver disease, coronary artery disease, cerebrovascular disease, uncontrolled hypertension, diabetes mellitus with vascular disease, or have had a stroke, heart attack, or blood clot within the past year, due to a possible reaction to Loestrin.
• Women who currently smoke cigarettes, due to the increased risk of serious cardiovascular events with Loestrin use.
Drug: Loestrin (norethindrone acetate and ethinyl estradiol)
Cystic Fibrosis (CF)
Cystic Fibrosis, Loestrin, Hormone
UT Southwestern; Children’s Health
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The iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study

This research study is evaluating the use of specialized testing of solid tumors including sequencing. The process of performing these specialized tests is called tumor profiling. The tumor profiling may result in identifying changes in genes of the tumor that indicate that a particular therapy may have activity. This is called an individualized cancer therapy (iCat) recommendation. The results of the tumor profiling and, if applicable, the iCat recommendation will be returned.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Laura Klesse
13954
All
up to 30 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02520713
STU 072015-038
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Inclusion Criteria:

• Age -- Age ≤ 30 years at time of initial qualifying solid tumor diagnosis
• Diagnosis -- Histologic diagnosis of solid malignancy (excluding brain tumors and lymphoma) that meets at least one of the following criteria:
• Refractory, defined as tumor progression after initiation of standard first line therapy without having achieved a prior partial or complete remission OR Biopsy proven residual disease at the completion of planned standard initial front-line therapy.
• Recurrent, defined as tumor progression after achieving a prior partial or complete remission
• Newly diagnosed high risk disease, defined as having an expected event free survival of < 50% at 2 years.
• Lacks definitive diagnosis or classical genomic findings after histologic review and standard molecular testing (rare tumor group).
• Examples include (eligibility not limited to these examples):
• Histology typically associated with a fusion in which fusion is not detected.
• Ewing-like sarcoma
• Undifferentiated sarcoma
• Inflammatory myofibroblastic tumor without ALK fusion
• Infantile fibrosarcoma without NTRK fusion
• Specimen Samples
• Sufficient tumor specimen available to meet the minimum requirements for profiling from diagnosis or progression / recurrence --- OR
• Surgery / biopsy planned as part of clinical care that is anticipated to yield sufficient material to meet the minimum requirements for profiling; OR
• Patient has already had molecular profiling and patient has not yet started matched targeted therapy based on the report .
Exclusion Criteria:

• No Therapy Planned -- Patients who have declined further anticancer therapy will be excluded.
• Performance Status -- Patients with Lansky (age < 16 years) or Karnofsky (age ≥16 years) score < 50 will be excluded.
• Life Expectancy -- Patients with anticipated life expectancy < 3 months will be excluded.
Genetic: Genetic testing and GAIN report
Sarcoma, Pediatric Solid Tumor, Brain and Nervous System, Other, Eye and Orbit, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Female, Breast - Male, Carcinoid Tumor, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Heart, Kaposis sarcoma, Small Intestine, Soft Tissue, Ill - Defined Sites
Pediatric Solid Tumor
Children’s Health
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Sigh Ventilation to Increase Ventilator-Free Days in Victims of Trauma at Risk for Acute Respiratory Distress Syndrome (SiVent)

A randomized, concurrent controlled trial to assess if adding sigh breaths to usual invasive mechanical ventilation of victims of trauma who are at risk of developing ARDS will decrease the number of days they require invasive mechanical ventilation.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Katherine.Bosler@UTSouthwestern.edu
Joseph Minei
14988
All
18 Years to 89 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02582957
STU 102015-009
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Inclusion Criteria:
Patients in an intensive care unit (ICU) as a result of injuries resulting from penetrating or non-penetrating trauma who are intubated and receiving invasive mechanical ventilation who also have one or more of the following: 1. Traumatic brain injury 2. > 1 long bone fractures 3. Shock on arrival in the Emergency Department (systolic BP < 90 mmHg) 4. Lung contusion 5. Receipt of > 6 units of blood
Exclusion Criteria:
1. Inability to obtain consent from the patient or his/her legally authorized representative (LAR) 2. Unwillingness of the treating physician to use sigh ventilation as all treating physicians must have equipoise with respect to the intervention 3. Age limitations per Institutional Review Board regulations 4. Undergoing invasive mechanical ventilation for > 24 hours, excluding any time during which the patient was being ventilated in the operating room, CT or IR, as this could represent too long a delay in instituting the intervention for it to have a chance of being effective 5. Presence of malignancy or other irreversible disease or condition for which the six month mortality is estimated to exceed 50% (e.g., chronic liver disease with a Child-Pugh Score of 10-15, malignancy refractory to treatment) as this could affect the clinical course and cloud interpretation of the endpoints 6. Women who are pregnant (negative pregnancy tests required on women of child-bearing age) per Human Subjects regulations 7. Prisoners, per Human Subjects regulations 8. Neurological condition that could impair spontaneous ventilation (e.g., C5 or higher spinal cord injury as this could affect the clinical course and cloud interpretation of the ventilator-free day endpoint 9. Lack of availability of Dräger Evita Infinity V500 ventilator as this is the only ventilator capable of delivering sigh breaths as described in the protocol 10. Burns > 40% of body surface area as this could affect the clinical course and cloud interpretation of the endpoints 11. Treating physicians being unwilling to use low VT ventilation strategy when ARDS is diagnosed as low VT ventilation is now considered standard of care for patients with ARDS. 12. Moribund, not expected to survive 24 hours as this could affect the clinical course and cloud interpretation of the endpoints13. Treating physician's decision to use airway pressure release ventilation (APRV). 13. Patient not expected to require mechanical ventilation > 24 hours (e.g., intubated for alcohol intoxication rather than pulmonary problem).
Other: Sigh breaths
Acute Respiratory Distress Syndrome
ARDS, Shock Lung, Acute Chest Syndrome, Respiratory Distress Syndrome, Adult, Respiratory insufficiency, Trauma
Parkland Health & Hospital System
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STaph Aureus Resistance-Treat Early and Repeat (STAR-TER) (STAR-TER)

To evaluate the micro-biologic efficacy and safety of a streamlined treatment for early onset methicillin-resistant staphylococcus aureus (MRSA) in patients with cystic fibrosis.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Mary.Klosterman@UTSouthwestern.edu
Preeti Sharma
117060
All
2 Years to 45 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03489629
STU 022018-089
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Inclusion Criteria:
1. Male or female ≥ 2 and ≤ 45 years of age at the Screening Visit. 2. Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria: 1. sweat chloride ≥ 60 milliequivalents/liter by quantitative pilocarpine iontophoresis test (QPIT) 2. two well-characterized mutations in the cystic fibrosis transmembrane conductive regulator (CFTR) gene 3. abnormal nasal potential difference(NPD) (change in NPD in response to a low chloride solution and isoproteronol of less than -5 mV) 3. First OR early MRSA colonization defined as: 1. First MRSA colonization: first documented isolation of MRSA from respiratory tract occurred ≤ 6 months prior to screening 2. Early MRSA colonization: MRSA was previously isolated from the respiratory tract ≤ 2 times over the past 3.5 years, but this was followed by at least 1 year of documented negative cultures for MRSA 4. MRSA is available to the central laboratory
•either the incident MRSA isolate from the clinic visit or the subject is MRSA positive at the screening visit 5. Clinically stable with no significant changes in health status within the 14 days prior to screening 6. 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. Received antibiotics with activity against MRSA within 28 days prior to screening 2. Use of an investigational agent within 28 days prior to screening 3. For subjects ≥ 6 years of age: FEV1 at screening < 25% of predicted for age based on the Wang (males < 18 years, females < 16 years) or Hankinson (males ≥ 18 years, females ≥ 16 years) standardized equations 4. MRSA from the screening culture or the most recent clinical care visit within 6 months prior to screening resistant to TMP/SMX 5. History of intolerance to topical chlorhexidine or mupirocin 6. History of intolerance to both TMP/SMX and minocycline 7. < 8 years of age and allergic or intolerant to TMP/SMX 8. ≥ 8 years of age and allergic or intolerant to TMP/SMX and MRSA isolate (from screening or clinical care visit)is resistant to minocycline 9. For females of child bearing potential: pregnant, breastfeeding, or unwilling to use barrier contraception through Day 42 of the study 10. Subjects with history of abnormal renal function will need screening labs showing normal function Abnormal renal function is defined as estimated creatinine clearance <50 mL/min using the: 1. Bedside Schwartz Equation for subjects <18 years of age, and 2. Levey Glomerular filtration rate (GFR) Equation for subjects ≥ 18 years of age. 11. Subjects with a history of abnormal liver function will need to have screening labs showing normal transaminases. Liver dysfunction is defined as ≥3x upper limit of normal (ULN), of serum aspartate transaminase (AST) or serum alanine transaminase (ALT) or abnormal synthetic function 12. History of solid organ or hematological transplantation 13. 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.
Drug: Trimethoprim Sulfamethoxazole (TMP/SMX), Drug: Minocycline, Drug: Mupirocin, Drug: Chlorhexidine Gluconate, Behavioral: Environmental Decontamination
Cystic Fibrosis
Methicillin-resistant Staphylococcus aureus (MRSA), Early infection, Treatment, Forced Expiratory Volume in 1 Second (FEV1)
Children’s Health
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A Study to Assess Pulsed Inhaled Nitric Oxide in Subjects With Pulmonary Fibrosis at Risk for Pulmonary Hypertension (REBUILD)

A randomized, double-blind, placebo-controlled dose escalation and verification study to assess the safety and efficacy of pulsed inhaled nitric oxide (iNO) in subjects at risk for pulmonary hypertension associated with pulmonary fibrosis on long term oxygen therapy (Part 1 and Part 2) - REBUILD
Call 214-648-5005
studyfinder@utsouthwestern.edu, Mary.Martin@UTSouthwestern.edu
Edward Mims
194632
All
18 Years to 80 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03267108
STU 102017-024
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Inclusion criteria:
• A high resolution CT scan performed in the 6 months prior to screening associated with one of the following conditions and confirmed using guidelines, as per American Thoracic Society (ATS) / European Respiratory Society (ERS) / Japanese Respiratory Society (JRS) / Latin American Thoracic Association (ALAT):
• Major IIPs (idiopathic interstitial pneumonias) diagnosis or suspected as one of the following:
• Idiopathic pulmonary fibrosis
• Idiopathic nonspecific interstitial pneumonia
• Respiratory bronchiolitis-interstitial lung disease
• Desquamative interstitial pneumonia
• Cryptogenic organizing pneumonia
• Acute interstitial pneumonia
• Rare IIPs diagnosis by one of the following:
• Idiopathic lymphoid interstitial pneumonia
• Idiopathic pleuroparenchymal fibroelastosis
• Unclassifiable idiopathic interstitial pneumonias
• Chronic hypersensitivity pneumonitis
• Occupational lung disease
• Connective Tissue Disease associated with IPF (CTD-ILD)
• Interstitial Pneumonia with Autoimmune Features (IPAF)
• Have been using oxygen therapy by nasal cannula for at least 4 weeks (including use limited to exertion)
• 6MWD ≥ 100 meters and ≤ 400 meters at screening and Baseline/Randomization visits.
• World Health Organization (WHO) Functional Class II-IV
• Forced Vital Capacity ≥ 40% predicted within the 60 day Screening period
• Age between 18 and 80 years (inclusive) at screening Exclusion criteria:
• For women of child-bearing potential: Pregnant or breastfeeding females at Screening, or planning to get pregnant, or unwilling to use appropriate contraception if sexually active to avoid pregnancy during the study and for at least 30 days after discontinuation of the study drug.
• Heart failure with reduced ejection fraction (HFrEF; systolic heart failure) with an ejection fraction of < 40%; or severe HF with preserved EF (HFpEF; diastolic HF)
• History of sarcoidosis
• History of chronic thrombo-embolic pulmonary hypertension (CTEPH; WHO group 4 PH)
• Smoking within 3 months of Screening and/or unwilling to avoid smoking throughout the study
• Body mass index (BMI) >40 kg/m2 at screening
• Intermittent uncontrolled atrial fibrillation, in the opinion of the Principal Investigator
• Known severe hepatic impairment, in the opinion of the Principal Investigator
• Known severe renal impairment (Chronic Kidney Disease Epidemiology Collaboration (CKDEPI) 2009 equation [Levy 2009] calculated creatinine clearance <30 ml/min) at screening
Combination Product: INOpulse®, Combination Product: Placebo, Combination Product: Open Label Extension
Pulmonary Hypertension, Pulmonary Fibrosis
Pulmonary Fibrosis, PF, Pulmonary Hypertension, Inhaled Nitric Oxide, iNO, Long Term Oxygen Therapy, Oxygen, REBUILD, INOpulse, pulsed inhaled nitric oxide, fILD, fibrotic interstitial lung disease, portable pulsed inhaled nitric oxide
UT Southwestern
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A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients

Study ROR-PH-301, ADVANCE OUTCOMES, is designed to assess the efficacy and safety of ralinepag when added to pulmonary arterial hypertension (PAH) standard of care or PAH-specific background therapy in subjects with World Health Organization (WHO) Group 1 PAH.
Call 214-648-5005
studyfinder@utsouthwestern.edu, tatyana.ganz@utsouthwestern.edu
Sonja Bartolome
115047
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03626688
STU 062018-068
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Inclusion Criteria:
1. At least 18 years of age. 2. Evidence of a personally signed and dated informed consent form indicating that the subject has been informed of all pertinent aspects of the study prior to initiation of any study-related procedures. 3. Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures 4. Primary diagnosis of symptomatic PAH. 5. Has had a right heart catheterization (RHC) performed at or within 3 years prior to Screening (RHC will be performed during Screening if not available) that is consistent with the diagnosis of PAH. 6. Has WHO/ NYHA functional class II to IV symptoms. 7. If on PAH-specific background oral therapy, subject is on stable therapy with either an endothelin receptor antagonist (ERA) and/or a phosphodiesterase type 5 inhibitor (PDE5-I) or a soluble guanylate cyclase (sGC) stimulator. Subjects may be naïve to PAH-specific treatment. 8. Has a 6MWD of ≥150 meters. 9. If taking concomitant medications that may affect the clinical manifestations of PAH (eg, calcium channel blockers, diuretics, digoxin, or L arginine supplementation, beta blockers, angiotensin-converting enzyme inhibitors, or angiotensin II receptor blockers), must be on a stable dose for at least 30 days prior to the Baseline Visit and the dosage maintained throughout the study. The exception is that the dose of diuretics must be stable for at least the 10 days prior to Baseline. 10. Both male and female subjects agree to use a highly effective method of birth control throughout the entire study period from informed consent through to the 30-Day Follow-up Visit, if the possibility of conception exists. Eligible male and female subjects must also agree not to participate in a conception process during the study and for 30 days after the last dose of IMP. Eligible male subjects must agree not to participate in sperm donation for 90 days after the last dose of IMP.
Exclusion Criteria:
1. For subjects with known HIV-associated PAH, a cluster designation 4 (CD4+) T-cell count <200/mm3 within 90 days of Baseline. 2. Must not have 3 or more left ventricular dysfunction risk factors as defined in the study protocol. 3. Has evidence of more than mild lung disease on pulmonary function tests performed within 180 days prior to, or during Screening. 4. Has evidence of thromboembolic disease as determined by a V/Q lung scan or local standard of care diagnostic evaluation at or after diagnosis of PAH. 5. Current diagnosis of ongoing and clinically significant sleep apnea as defined by the Investigator. 6. Male subjects with a corrected QT interval using Fridericia's formula (QTcF) >450 msec and female subjects with a QTcF >470 msec on ECG recorded at Screening and analyzed by the central ECG laboratory. Subjects with evidence of intraventricular conduction delay, defined as a QRS interval greater than 110 msec, will be excluded if the QTcF is >500 msec for both males and females. 7. Severe chronic liver disease (ie, Child-Pugh Class C), portal hypertension, cirrhosis or complications of cirrhosis/portal hypertension (eg, history of variceal hemorrhage, encephalopathy). 8. Confirmed active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV). 9. Subjects with alanine aminotransferase or aspartate aminotransferase ≥3 times the upper limit of normal (ULN) or total bilirubin ≥2 × ULN at Screening. 10. Chronic renal insufficiency as defined by serum creatinine >2.5 mg/dL or requiring dialysis at Screening. 11. Hemoglobin concentration <9 g/dL at Screening. 12. Subjects treated with an IV or SC prostacyclin pathway agent (eg, epoprostenol, treprostinil, or iloprost) for PAH at any time prior to Baseline (use in vasoreactive testing is permitted). 13. Subjects currently on or who were treated with an inhaled or oral prostacyclin pathway agent (iloprost, treprostinil, beraprost, or selexipag) within 90 days prior to Baseline. 14. Subject has pulmonary veno-occlusive disease. 15. Malignancy diagnosed and/or treated within 5 years prior to Screening, with the exception of localized non-metastatic basal cell or squamous cell carcinoma of the skin or in-situ carcinoma of the cervix excised with curative intent. 16. Subject tests positive for amphetamine, cocaine, methamphetamine, methylenedioxymethamphetamine or phencyclidine in urine drug screen performed at Screening, or has a recent history (6 months) of alcohol or drug abuse. A subject will not be excluded due to a positive drug screen caused by prescribed medications. 17. Initiation or discontinuation of a cardio-pulmonary rehabilitation program based upon exercise within 90 days prior to Screening and/or planned during study participation. 18. Prior participation in any study of ralinepag or participation in another interventional clinical study with medicinal products within 30 days prior to Screening. Concurrent participation in registry or observational studies is allowed, as long as the subject can fulfill all other entry criteria and comply with all study procedures. 19. Any reason that, in the opinion of the Investigator or Medical Monitor, precludes the subject from participating in the study (eg, any previous or intercurrent medical condition) that may increase the risk associated with study participation or that would confound study analysis or impair study participation or cooperation. 20. Known hypersensitivity to ralinepag or any of the excipients. 21. Life expectancy <12 months based on the Investigator's opinion. 22. Women who are pregnant, lactating or breast-feeding.
Drug: Ralinepag, Drug: Placebo
Pulmonary Hypertension, Pulmonary Arterial Hypertension, Hypertension, Cardiovascular Diseases, Hypertension, Pulmonary, PAH, Connective Tissue Diseases, Familial Primary Pulmonary Hypertension, Vascular Diseases, Lung Diseases, Respiratory Tract Disease, Lung/Thoracic
Prostacyclin, Connective Tissue Disease-Associated, 6 Minute Walk Test, 6 Minute Walk Distance, Pulmonary Vascular Resistance, Right Ventricular Function
UT Southwestern
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PROSpect: Prone and Oscillation Pediatric Clinical Trial

Severe pediatric acute respiratory distress syndrome (PARDS) is a life-threatening and frequent problem experienced by thousands of children each year. Little evidence supports current supportive practices during their critical illness. The overall objective of this study is to identify the best positional and/or ventilation practice that leads to improved patient outcomes in these critically ill children. We hypothesize that children with severe PARDS treated with either prone positioning or high-frequency oscillatory ventilation (HFOV) will demonstrate more days off the ventilator when compared to children treated with supine positioning or conventional mechanical ventilation (CMV).
Call 214-648-5005
studyfinder@utsouthwestern.edu, Morgan.Rogers@childrens.com
Peter Luckett
14466
All
up to 18 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03896763
STU-2019-0488
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Inclusion criteria: Intubated and mechanically ventilated with moderate-severe PARDS for <48 hours per PALICC guidelines (chest imaging consistent with acute pulmonary parenchymal disease and OI ≥12 or OSI ≥10). We require two blood gases meeting moderate-severe PARDS criteria (separated by at least 4 ± 2 hours during which time the clinical team is actively working to recruit lung volume and optimize the patient's hemodynamic status per PALICC guidelines; specifically, incremental and decremental PEEP changes to optimize lung volume). A second blood gas is not required for OI ≥16. Exclusion criteria:
• Perinatal related lung disease
• Congenital diaphragmatic hernia or congenital/acquired diaphragm paralysis
• Respiratory failure explained by cardiac failure or fluid overload
• Cyanotic heart disease
• Cardiomyopathy
• Unilateral lung disease
• Primary pulmonary hypertension
• Intubated for status asthmaticus
• Obstructive airway disease (e.g., Severe airways disease without parenchymal involvement or disease characterized by hypercapnia with FiO2 <0.30 and/or evidence of increased resistance visible on the flow
•time scalar and/or presence of intrinsic PEEP)
• Active air leak
• Bronchiolitis obliterans
• Post hematopoietic stem cell transplant; specifically, patients receiving continuous supplemental oxygen for three or more days prior to intubation; receiving noninvasive ventilation for more than 24 hours prior to intubation; receiving more than one vasoactive medication at time of meeting inclusion criteria; spending more than four days in the PICU prior to intubation; supported on or with immediate plans for renal replacement therapies; with two or more allogeneic transplants; who relapsed after the transplant; or with diffuse alveolar hemorrhage
• Post lung transplant
• Home ventilator (including noninvasive) or home oxygen dependent (exception: night-time noninvasive ventilation (CPAP/BiPAP) or oxygen for obstructive sleep apnea is permitted)
• Neuromuscular respiratory failure
• Critical airway (e.g., post laryngotracheal surgery or new tracheostomy) or anatomical obstruction of the lower airway (e.g., mediastinal mass)
• Facial surgery or trauma in previous 2 weeks
• Head trauma (managed with hyperventilation)
• Intracranial bleeding
• Unstable spine, femur or pelvic fractures
• Acute abdominal process/open abdomen
• Morbid obesity (2w-24 months: WHO weight-for-length/height z-score ≥+3; ≥2 years: WHO body mass index (BMI)-for-age z-score ≥+3)
• Currently receiving either prone positioning or any high-frequency mode of MV with current illness (Up to 4 hours of prone positioning and/or any mode of high-frequency mode of MV is allowed as long as the therapies are off for least 4 hours prior to the subject meeting oxygenation criteria.)
• Supported on ECMO during the current admission
• Family/medical team not providing full support (patient treatment considered futile)
• Previously enrolled in current study
• Enrolled in any other interventional clinical trial not approved for co-enrollment
• Known pregnancy
Other: Either supine or prone positioning and either CMV or HFOV
Acute Respiratory Distress Syndrome in Children, Lung/Thoracic
Pediatric Acute Respiratory Distress Syndrome (PARDS), Acute Respiratory Distress Syndrome (ARDS), acute respiratory failure, child, pediatric intensive care unit
Children’s Health
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Clinical Study to Compare the Efficacy and Safety of Macitentan and Tadalafil Monotherapies With the Corresponding Fixed-dose Combination Therapy in Subjects With Pulmonary Arterial Hypertension (PAH) (A DUE)

Combination therapy in pulmonary arterial hypertension (PAH) has been the subject of active investigation for more than a decade, with the benefit of targeting different pathways known to be involved in the pathogenesis of the disease. Adherence to prescribed therapy has an impact on clinical outcomes. Reducing the pill/tablet count and frequency has a major impact on patients' adherence to therapies and therefore the observed clinical outcomes. One way to simplify treatment is to use fixed-dose combination (FDC) products that combine multiple treatments targeting different pathways into a single tablet. This study aims to demonstrate that the FDC of macitentan and tadalafil is more effective than therapy with 10 mg of macitentan alone or 40 mg of tadalafil alone. This phase 3 study will evaluate the efficacy and safety at 16 weeks of an FDC (macitentan 10 mg and tadalafil 40 mg) against these two PAH-approved therapies given as monotherapy to further confirm the added value of the FDC.
Call 214-648-5005
studyfinder@utsouthwestern.edu, tatyana.ganz@utsouthwestern.edu
Trushil Shah
169968
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03904693
STU-2020-0178
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Inclusion Criteria:

• Signed and dated informed consent form (ICF)
• Confirmed diagnosis of symptomatic PAH in WHO FC II or III
• Symptomatic PAH belonging to one of the following subgroups of WHO Group 1 pulmonary hypertension:
• Idiopathic
• Heritable
• Drug- or toxin-induced
• Associated with connective tissue disease, HIV infection, portal hypertension or congenital heart disease with simple systemic-to-pulmonary shunt with persistent pulmonary hypertension documented by a right heart catheterization (RHC) ≥ 1 year after surgical repair
• PAH diagnosis confirmed by hemodynamic evaluation at rest (through central reading), evaluated within 5 weeks prior to randomization:
• Mean pulmonary artery pressure (mPAP) ≥ 25 mmHg, AND
• Pulmonary artery wedge pressure (PAWP) or left ventricular end diastolic pressure (LVEDP) ≤ 15 mmHg, AND
• Pulmonary vascular resistance (PVR) ≥ 3 WU (i.e., ≥ 240 dyn∙sec∙cm-5)
• Negative vasoreactivity test in idiopathic, heritable, and drug/toxin-induced PAH. (Participants for whom no vasoreactivity test was performed at diagnosis can be eligible if currently treated with PAH therapy for more than 3 months and PAH diagnosis confirmed by hemodynamic evaluation at least 3 months after introduction of their PAH therapy).
• Currently receiving a stable dose of ERA or PDE-5i monotherapy for at least 3 months prior to baseline RHC, within the prespecified doses in the study protocol or no history of PAH-specific treatment
• Participant able to perform the 6MWT with a minimum distance of 100 m and maximum distance of 450 m at Screening
• A woman of childbearing potential must:
• have negative serum pregnancy test at Screening and a negative urine pregnancy test at Randomization
• agree to undertake monthly urine pregnancy tests during the study and up to at least 30 days after study treatment discontinuation
• agree to follow the contraception scheme from Screening up to at least 30 days after study treatment discontinuation
Exclusion Criteria:

• Treatment with a soluble guanylate cyclase stimulator, L-arginine, any form of prostanoids or prostacyclin-receptor agonists (including oral, inhaled, or infused routes) in the 3-month period prior to start of treatment
• Treatment with combination therapy of ERA and PDE-5i in the 3-month period prior to start of treatment or history of intolerance to ERA and PDE-5i combination therapy
• Hypersensitivity to any of the study treatments or any excipient of their formulations
• Treatment with a strong cytochrome P450 3A4 (CYP3A4) inducer in the 1-month period prior to start of treatment
• Treatment with a strong CYP3A4 inhibitor or a moderate dual CYP3A4/CYP2C9 inhibitor or co-administration of a combination of moderate CYP3A4 and moderate CYP2C9 inhibitors in the 1-month period prior to start of treatment
• Treatment with doxazosin
• Treatment with any form of organic nitrate, either regularly or intermittently
• Diuretic treatment initiated or dose changed within 1 week prior to the RHC or start of treatment
• Treatment with another investigational drug in the 3-month period prior to start of treatment
• Body mass index (BMI) > 40 kg/m2 at Screening
• Known presence of three or more of the following risk factors for heart failure with preserved ejection fraction at Screening:
• BMI > 30 kg/m2
• Diabetes mellitus of any type
• Essential hypertension (even if well controlled)
• Coronary artery disease, i.e. history of stable angina or known more than 50% stenosis in a coronary artery or history of myocardial infarction or history of or planned coronary artery bypass grafting and/or coronary artery stenting
• Known presence of moderate or severe obstructive lung disease any time prior to Screening as specified in study protocol
• Known presence of moderate or severe restrictive lung disease any time prior to Screening as specified in study protocol
• Clinically significant aortic or mitral valve disease; pericardial constriction; restrictive or congestive left-sided cardiomyopathy; life-threatening cardiac arrhythmias; significant left ventricular dysfunction; or left ventricular outflow obstruction, in the opinion of the investigator
• Known permanent atrial fibrillation, in the opinion of the investigator
• Known or suspected uncontrolled thyroid disease (hypo- or hyperthyroidism)
• Documented pulmonary veno-occlusive disease
• Hemoglobin < 100 g/L (<10 g/dL) at Screening
• Known severe hepatic impairment as specified in study protocol
• Serum aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 1.5 × upper limit of normal (ULN) at Screening
• Severe renal impairment at Screening as specified in study protocol
• Systemic hypotension at Screening or Randomization and systemic hypertension at Screening as specified in study protocol
• Acute myocardial infarction or cerebrovascular event (e.g., stroke) within the last 26 weeks prior to Screening
• Known bleeding disorder, in the opinion of the investigator
• Loss of vision in one or both eyes because of non-arteritic anterior ischemic optic neuropathy
• Hereditary degenerative retinal disorders, including retinitis pigmentosa
• History of priapism, conditions that predispose to priapism (example, sickle cell anemia, multiple myeloma, or leukemia) or anatomical deformation of the penis (example, angulation, cavernosal fibrosis, or Peyronie's disease)
• Difficulty swallowing large pills/tablets that would interfere with the ability to comply with study treatment regimen
• Any planned surgical intervention (including organ transplant) during the double-blind treatment period, except minor interventions
• Exercise training program for cardiopulmonary rehabilitation in the 12-week period prior to start of treatment, or planned to be started during the double-blind period of the study
• Pregnant, planning to become pregnant or lactating
• Any known factor or disease that might interfere with treatment adherence, full participation in the study or interpretation of the results as judged by the investigator (e.g., drug or alcohol dependence etc.)
• Known concomitant life-threatening disease with a life expectancy less than (<) 12 months
• Calcium channel blocker treatment initiated, or dose changed within 3 months prior to right heart catheterization (RHC) at screening
Drug: FDC macitentan/tadalafil, Drug: Macitentan 10 mg, Drug: Tadalafil 40 mg, Drug: Placebo FDC, Drug: Placebo macitentan, Drug: Placebo tadalafil
Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH), Cardiovascular, Lung/Thoracic
Pulmonary Arterial Hypertension, PAH, macitentan, tadalafil, fixed dose combination therapy
UT Southwestern
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