Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation

NCT03663933 | Active Not Recruiting Phase 2 Results DMC FDA Drug

• 2 other identifiers: 18013518-C-0135
• Study Type: interventional
• Target: 71
• Locations: 1 country
• Sites: 2

Brief Summary

Background: Blood stem cells in the bone marrow make all the cells to normally defend a body against disease. Allogeneic blood or marrow transplant is when these stem cells are transferred from one person to another. Researchers think this treatment can provide a new, healthy immune system to correct T-cell problems in some people. Objective: To see if allogeneic blood or bone marrow transplant is safe and effective in treating people with T-cell problems. Eligibility: Donors: Healthy people ages 4 and older Recipients: People the same age with abnormal T-cell function causing health problems Design: All participants will be screened with:

• Medical history
• Physical exam
• Blood, heart, and urine tests Donors will also have an electrocardiogram and chest x-ray. They may have veins tested or a pre-anesthesia test. Recipients will also have lung tests. Some participants will have scans and/or bone marrow collected by needle in the hip bones. Donors will learn about medicines and activities to avoid and repeat some screening tests. Some donors will stay in the hospital overnight and have bone marrow collected with anesthesia. Other donors will get shots for several days to stimulate cells. They will have blood removed by plastic tube (IV) in an arm vein. A machine will remove stem cells and return the rest of the blood to the other arm. Recipients will have:
• More bone marrow and a small fragment of bone removed
• Dental, diet, and social worker consultations
• Scans
• Chemotherapy and antibody therapy for 2 weeks
• Catheter inserted in a chest or neck vein to receive donor stem cells
• A hospital stay for several weeks with more medicines and procedures
• Multiple follow-up visits

Conditions

Lymphoproliferative Disorders; Autoimmune Lymphoproliferative; Primary T-cell Immunodeficiency Disorders; Immune System Diseases; Common Variable Immunodeficiency

Keywords

Haploidentical; Autoimmunity; Immune Dysregulation; Congenital; Opportunistic Infection

Outcome Measures

Primary Outcomes (2)

• Percentage of Recipients Who Are Alive With >50% Donor T Cell Chimerism and Graft-failure Free at 180 Days Post Hematopoietic Cell Transplant (HCT) Reported With an 80% Confidence Interval

  Percentage of recipients with \> 50% donor T cell chimerism and without death or graft failure. A graft failure event is defined as either primary or secondary graft failure, in the absence of a recurrent marrow malignancy.

  Day +180 post-HCT

• Percentage of Recipients Who Are Alive With >50% Donor T Cell Chimerism and Graft-failure Free at 180 Days Post Hematopoietic Cell Transplant (HCT) Reported With a 95% Confidence Interval

  Percentage of recipients with \> 50% donor T cell chimerism and without death or graft failure. A graft failure event is defined as either primary or secondary graft failure, in the absence of a recurrent marrow malignancy.

  Day +180 post -HCT

Secondary Outcomes (16)

• Cumulative Incidence of Transplant-related Mortality

  Day +180, and 1-year post-transplant

• Cumulative Incidence of Secondary Graft Failure

  1-, 3-, and 5-years post-transplant

• Percent Probability of Overall Survival (OS)

  1-, 3-, and 5-years post-transplant

• Percentage of Participants Who Achieve Chimerism at Stated Days Between Those Who Have Failed by Day 60 or Have Not

  Day +21, +28, +35, +42, and +60 after hematopoietic cell transplant (HCT)

• Percentage of Donor T-cell Populations at Days +28, +42, +60, +100, +180, and 1-year Post Hematopoietic Cell Transplant (HCT)

  Days +28, +42, +60, +100, +180, and 1-year post hematopoietic cell transplant

Other Outcomes (1)

• Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)

  Conditioning start until return to baseline/stabilization, 30 days post-therapy end, removal from therapy, or off study, whichever comes first for all AEs, followed by AE collection per principal investigator discretion, on average 2 years.

Study Arms (3)

Arm 1/Reduced-Intensity Conditioning (RIC)

EXPERIMENTAL

Reduced Intensity Conditioning Arm.

Drug: e-ATG; Procedure: Immunosuppression Only Conditioning; Procedure: Reduced Intensity Conditioning; Drug: GVHD Prophylaxis; Procedure: Allogeneic HSC; Drug: Bisulfan; Drug: Prednisone; Drug: Cyclophosphamide; Drug: MMF; Drug: Mesna; Drug: Tacrolimus; Drug: Pentostatin; Diagnostic Test: PFTs; Diagnostic Test: DEXA; Procedure: Bone Marrow Aspirate & Biopsy; Diagnostic Test: EKG; Diagnostic Test: 2D ECHO

Arm 2/Immunosuppression-only Conditioning (IOC)

EXPERIMENTAL

Immunosuppression Only Conditioning Arm.

Drug: e-ATG; Procedure: Immunosuppression Only Conditioning; Drug: GVHD Prophylaxis; Procedure: Allogeneic HSC; Drug: Bisulfan; Drug: Prednisone; Drug: Cyclophosphamide; Drug: MMF; Drug: Mesna; Drug: Tacrolimus; Drug: Pentostatin; Diagnostic Test: PFTs; Diagnostic Test: DEXA; Procedure: Bone Marrow Aspirate & Biopsy; Diagnostic Test: EKG; Diagnostic Test: 2D ECHO

Arm 3/Donor

NO INTERVENTION

Healthy Donor- Donors for recipients in arm 1 or arm 2.

Interventions

Reduced Intensity Conditioning PROCEDURE

Equine anti-thymocyte globulin (e-ATG) 40 mg/kg intravenous (IV) once daily for days -14 and -13. Prednisone: Tapering doses, given orally daily, and given prior to each daily dose of e-ATG on days -14 and -13, Pentostatin:4 mg/m\^2/day IV on days -11 and -7, cyclophosphamide: 5 mg/kg orally daily on days -11 through -4, Busulfan IV, pharmacokinetically dosed, on days -3 and -2.

Also known as: RIC

Arm 1/Reduced-Intensity Conditioning (RIC)

PFTs DIAGNOSTIC_TEST

Screening ≤4 weeks pretreatment (rx), Day +180 (≤ 14 days), Day +36 (± 21 days), Day +548 (18 months) (± 28 days), and at 2 years and yearly thereafter through +5 years (± 56 days).

Also known as: Pulmonary function tests

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

Bone Marrow Aspirate & Biopsy PROCEDURE

Baseline, Day +60 (± 3 days) and Day +365 (±21 days).

Also known as: BM aspirate & bx

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

EKG DIAGNOSTIC_TEST

Baseline

Also known as: Electrocardiogram

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

2D ECHO DIAGNOSTIC_TEST

Screening ≤4 weeks pretreatment (rx), Day +180 (≤ 14 days), Day +36 (± 21 days), Day +548 (18 months) (± 28 days), and at 2 years and yearly thereafter through +5 years (± 56 days).

Also known as: 2-dimensional echocardiogram

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

e-ATG DRUG

During Immunosuppression Only Conditioning (IOC) and Reduced Intensity Conditioning (RIC).

Also known as: Antithymocyte Globulin (Equine), Atgam

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

Immunosuppression Only Conditioning PROCEDURE

Equine anti-thymocyte globulin (e-ATG) 40 mg/kg intravenous (IV) once daily for days -14 and -13. Prednisone: Tapering doses, given orally daily, and given prior to each daily dose of e-ATG on days -14 and -13, Pentostatin:4 mg/m\^2/day IV on days -9 and -5, cyclophosphamide:5 mg/kg orally daily on days -9 through -2.

Also known as: IOC

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

GVHD Prophylaxis DRUG

High-dose, post-transplantation cyclophosphamide (PTCy) 25-50 mg/kg on days +3 and +4, Mesna: 25-50 mg/kg weight-based dosing, Tacrolimus 0.02 mg/kg on days +5 through +90, and mycophenolate mofetil (MMF) 15 mg/kg on days +5 through +25.

Also known as: Graft-versus-host disease prophylaxis

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

Allogeneic HSC PROCEDURE

Stem cell transplant

Also known as: Allogeneic hematopoietic stem cell (HSC)

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

Bisulfan DRUG

During Reduced Intensity Conditioning (RIC).

Also known as: Busulfex, Myleran

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

Prednisone DRUG

During Immunosuppression Only Conditioning (IOC) and Reduced Intensity Conditioning (RIC).

Also known as: Deltasone

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

Cyclophosphamide DRUG

During Immunosuppression Only Conditioning (IOC), Reduced Intensity Conditioning (RIC) and Graft-versus-host disease prophylaxis (GVHD).

Also known as: Cytoxan, Neosar

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

MMF DRUG

During Graft-versus-host disease prophylaxis (GVHD).

Also known as: Mycophenolate mofetil, CellCept

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

Mesna DRUG

During Graft-versus-host disease prophylaxis (GVHD).

Also known as: Mesnex, Uromitexan

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

Tacrolimus DRUG

During Graft-versus-host disease prophylaxis (GVHD).

Also known as: Prograf

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

Pentostatin DRUG

During Immunosuppression Only Conditioning (IOC) and Reduced Intensity Conditioning (RIC).

Also known as: Nipent, Deoxycoformycin

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

DEXA DIAGNOSTIC_TEST

Baseline, Day +365 (± 21 days), at 2 years and yearly thereafter through +5 years (± 56 days), and as clinically indicated after hematopoietic cell transplant (HCT).

Also known as: Dual-energy x-ray absorptiometry

Arm 1/Reduced-Intensity Conditioning (RIC); Arm 2/Immunosuppression-only Conditioning (IOC)

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \>= 4 years
• T-cell proliferation and/or dysregulation (TCP/D) deemed to be of sufficient past severity to warrant hematopoietic cell transplantation (HCT) that meets at least one of the criteria below:
• Identified germline T-cell activating mutation in the phosphoinositide 3-kinase (PI3k) pathway
• Identified adenosine deaminase 2 (ADA2) deficiency (biallelic mutations in CECR1 (ADA2) and/or phenotypically with low ADA2 level) leading to neutropenia requiring chronic granulocyte colony-stimulating factor (GCSF) therapy or to transfusion-dependent anemia or thrombocytopenia
• T-cell infiltration of liver, spleen, lymph nodes, marrow, lungs, gut, or other organs by T cells, as evidenced by laboratory, radiographic, and/or anatomic pathology evaluation, resulting in organ dysfunction and/or organomegaly
• Latent herpesvirus infection in T lymphocytes
• History of or active evidence of hemophagocytic lymphohistiocytosis (HLH)
• Recurrent or prolonged fevers attributed to immune dysregulation
• T-cell population in blood and/or marrow with immunophenotype of large granular lymphocytes (LGL), with or without clonality or lymphocytosis
• T-cell lymphoproliferative disorder in the setting of an underlying immune defect
• Immune-mediated cytopenias of one lineage requiring transfusion or GCSF support or of 2 or 3 lineages with or without transfusion or support
• Chronic active Epstein-Barr virus (EBV)
• At least one potential 7-8/8 human leukocyte antigen (HLA)-matched related (excluding an identical twin) or unrelated donor (at HLA-A, -B, -C, and -DR), or an HLA-haploidentical related donor, based on initial low resolution unrelated donor search and/or at least one biologically- related family member who has at least a 25% chance of being at minimum an HLA- haploidentical match and is potentially suitable to donate based on reported family history. HLA typing of potential donors and/or mutation testing does not need to be completed for eligibility.
• Adequate end-organ function, as measured by:
• Left ventricular ejection fraction (LVEF) greater than or equal to 40% by 2-dimensional (2D) echocardiogram (ECHO) or left ventricular shortening fraction greater than or equal to 20% by ECHO for subjects receiving reduced-intensity conditioning (RIC), or LVEF greater than or equal to 30% if the subject has radiologic evidence of aortic, renal, or coronary artery vasculitis. LVEF greater than or equal to 30% for subjects receiving immunosuppression-only conditioning (IOC).

You may not qualify if:

• Subjects who are receiving any other investigational agents, with the exception of virus- specific cytotoxic T-cells for the treatment of viral infection/reactivation prior to allo HCT.
• Prohibitive allergy to a study drug or to compounds of similar chemical or biologic composition of the agents (equine anti-thymocyte globulin (e-ATG), steroids, cyclophosphamide, busulfan, pentostatin, tacrolimus, mycophenolate mofetil (MMF), G-CSF) used in the study.
• Active psychiatric disorder which is deemed by the PI to have significant risk of compromising compliance with the transplant protocol, or which does not allow for appropriate informed consent
• Human immunodeficiency virus (HIV) positive or other acquired immunodeficiency that, as determined by the PI, interferes with the assessment of TCP/D severity and/or the attribution of clinical manifestations of immunodeficiency to a disorder of TCP/D.
• Magnesium Transporter 1 (MagT1) mutation and active need to take anti-platelet agents and/or therapeutic anti- coagulation that cannot be interrupted during aplasia
• Lack of adequate central venous access potential
• Age greater than or equal to 4 years
• Related donor deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood, urine, and marrow specimens for research. Related donors will be evaluated in accordance with existing Standard Policies and Procedures for determination of eligibility and suitability for clinical donation. Note that participation in this study is offered to all related donors, but is not required for clinical donation, so it is possible that not all related donors will enroll onto this study.
• None
• Unrelated donors will be evaluated in accordance with existing National Marrow Donor Program (NMDP) Standard Policies and Procedures, available at: http://bethematch.org/About-Us/Global- transplant-network/Standards/, except for the additional requirement of EBV serostatus testing for clinical purposes of donor selection. Note that participation in this study is offered to all unrelated donors but not required for clinical donation, so it is possible that not all unrelated donors will enroll on this study. Unrelated donors only enroll if they contribute research specimens, which is optional.
• Unrelated donors: failure to qualify as a National Marrow Donor Program (NMDP) donor per current NMDP Standards, available at: http://bethematch.org/About-Us/Global-transplant-network/Standards/. Exceptions to donor eligibility (e.g. foreign travel, tattoos) do not automatically exclude the donor and will be reviewed by the PI.

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (2)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

National Marrow Donor Program

Minneapolis, Minnesota, 55401, United States

Location

Related Publications (9)

• Dimitrova D, et al. BK virus-associated hemorrhagic cystitis in posttransplant cyclophosphamide-based allogeneic hematopoietic cell transplantation (HCT) for immune deficiency or dysregulation. The 50th Annual Meeting of the European Society for Blood and Marrow Transplantation; Glasgow, United Kingdom; April 16, 2024.

  BACKGROUND

• Cusmano A, Soldatos A, Notarangelo L, Grimes A, Makkeyah S, Selim L, Kanakry J, Dimitrova D. Allogeneic hematopoietic cell transplantation (HCT) in two pediatric patients with central nervous system-restricted familial hemophagocytic lymphohistiocytosis. The 50th Annual Meeting of the European Society for Blood and Marrow Transplantation; Glasgow, United Kingdom; April 15, 2024. (presenting and senior author)

  BACKGROUND

• Cusmano A, Maher J, Gomez-Lobo V, Freeman A, Uzel G, Kanakry JA, Dimitrova D. Ovarian function following reduced intensity conditioning (RIC) allogeneic hematopoietic cell transplantation (HCT): how fertile is the future? Transplantation and Cellular Therapy Meetings of ASTCT and CIBMTR; San Antonio, TX; February 22, 2024. (presenting and senior author)

  BACKGROUND

• Dimitrova D, et al. Pre-Transplant Anti-CD20 Monoclonal Antibody Therapy Affects the Donor-Derived Hematopoietic Compartment in Allogeneic Hematopoietic Cell Transplantation (HCT) Recipients. The 49th Annual Meeting of the European Society for Blood and Marrow Transplantation; Paris, France; April 23-26, 2023.

  BACKGROUND

• Dimitrova D, et al. Humoral Reconstitution after Allogeneic Hematopoietic Cell Transplantation (HCT) in Patients Pretreated with Targeted Anti-CD20 Therapy. Transplantation and Cellular Therapy Meetings of ASTCT and CIBMTR; Orlando, FL; February 16, 2023.

  BACKGROUND

• Dimitrova D, Napier S, Stokes A, Uzel G, Miljkovic M, Pittaluga S, Wang H, Notarangelo LD, Ombrello AK, Stone D, Cuellar-Rodriguez J, Wilder J, Hicks SN, Sadler JL, Fowler DH, Gress RE, Kanakry CG, Kanakry JA. Distal Equine Anti-Thymocyte Globulin (ATG) As an Adjunct to Reduced Intensity Conditioning and Posttransplantation Cyclophosphamide (PTCy) for Allogeneic Hematopoietic Cell Transplantation (HCT) in Patients with Benign and Malignant Disorders of T Cell Proliferation or Dysregulation. Transplantation and Cellular Therapy Meetings of ASTCT and CIBMTR; virtual; February 8-12, 2021.

  BACKGROUND

• Dimitrova D, Uzel G, Notarangelo LD, Ombrello AK, Stone D, Parta M, Carroll E, Wilder J, Hicks SN, Sadler JL, Fowler DH, Gress RE, Kanakry CG, Kanakry JA. Novel Reduced Intensity Conditioning (RIC) Approach to Allogeneic Hematopoietic Cell Transplantation (HCT) in Patients with Benign and Malignant Disorders of T Cell Proliferation or Dysregulation. The 46th Annual Meeting of the European Society for Blood and Marrow Transplantation; virtual; August 2020.

  BACKGROUND

• Rechache, Kamil & Dimitrova, Dimana & Feng, Xingmin & Flomerfelt, Francis & Napier, Scott & Sponaugle, Jennifer & Stokes, Anita & Hyder, Mustafa & McKeown, Christi & Wilder, Jennifer & Baruffaldi, Judy & Chai, Amy & Walker, Melissa & Gress, Ronald & Kanakry, Christopher & Kanakry, Jennifer. (2022). Distally-Timed Equine Antithymocyte Globulin (eATG) in Allogeneic Hematopoietic Cell Transplantation (HCT) Conditioning - Pharmacokinetics and the Relationship between Total E-ATG Levels, Pre-HCT Absolute Lymphocyte Count, Immune Reconstitution, and Graft-Versus-Host Responses. Transplantation and Cellular Therapy. 28. S80. 10.1016/S2666-6367(22)00254-8.

  BACKGROUND

• Kamil A. Rechache, Natalia S. Nunes, Xingmin Feng, Francis A Flomerfelt, William Telford, Brian Dawson, Thomas E. Hughes, Syed Muhammad Salman Shah, Jennifer Sponaugle, Amy Chai, Jessenia Campos, Mustafa A. Hyder, Dimana Dimitrova, Christopher G. Kanakry, Jennifer A. Kanakry,The Pharmacokinetics and Pharmacodynamics of Distally-Timed Eatg in Allogeneic Hematopoietic Cell Transplantation Conditioning,Transplantation and Cellular Therapy,Volume 31, Issue 2, Supplement,2025,Pages S174-S175,ISSN 2666-6367,https://doi.org/10.1016/j.jtct.2025.01.269.

  BACKGROUND

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Lymphoproliferative Disorders; T cell immunodeficiency primary; Immune System Diseases; Common Variable Immunodeficiency; Autoimmune Diseases; Opportunistic Infections

Interventions

Antilymphocyte Serum; Busulfan; Prednisone; Cyclophosphamide; Mycophenolic Acid; Mesna; Tacrolimus; Pentostatin; Respiratory Function Tests; Absorptiometry, Photon; Biopsy; Electrocardiography

Condition Hierarchy (Ancestors)

Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immunologic Deficiency Syndromes; Infections

Intervention Hierarchy (Ancestors)

Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures; Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Sulfhydryl Compounds; Macrolides; Lactones; Coformycin; Formycins; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Deoxyribonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Diagnostic Techniques, Respiratory System; Diagnostic Techniques and Procedures; Diagnosis; Radiography; Diagnostic Imaging; Densitometry; Photometry; Chemistry Techniques, Analytical; Investigative Techniques; Cytodiagnosis; Cytological Techniques; Clinical Laboratory Techniques; Specimen Handling; Diagnostic Techniques, Surgical; Surgical Procedures, Operative; Heart Function Tests; Diagnostic Techniques, Cardiovascular; Electrodiagnosis

Results Point of Contact

• Title: Dr. Dimana Dimitrova
• Organization: National Cancer Institute

dimana.dimitrova@nih.gov

240-858-3647

Study Officials

• Dimana Dimitrova, M.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: NIH
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 7, 2018
• First Posted: September 10, 2018
• Study Start: September 4, 2018
• Primary Completion: April 3, 2025
• Study Completion (Estimated): April 3, 2030
• Last Updated: March 17, 2026
• Results First Posted: March 17, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Clinical data available during the study and indefinitely. Genomic data is available once genomic data is uploaded per protocol Genomic Data Sharing (GDS) plan for as long as database is active.
• Access Criteria: Clinical data will be made available with the permission of the study principal investigator (PI). Genomic data is made available via the database of Genotypes and Phenotypes (dbGaP) through requests to the data custodians.

Locations

US (2)