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ClinicalTrials.gov public records Last synced May 21, 2026, 7:29 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Severe Combined Immunodeficiency, X Linked Clear all
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Chronic Granulomatous Disease (CGD), X-Linked Severe Combined Immune Deficiency (XSCID), Leukocyte Adhesion Deficiency 1 (LAD), Graft Versus Host Disease (cGvHD)
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
3,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced May 21, 2026, 7:29 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Stem cell transplant, Filgrastim, Alemtuzumab, Miltenyi CliniMACS
Procedure · Drug · Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 2 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 19, 2009 · Synced May 21, 2026, 7:29 PM EDT
Suspended Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Severe Combined Immunodeficiency Disease, X-linked
Interventions
CL20-i4-EF1α-hγc-OPT, Busulfan, CliniMacs
Genetic · Drug · Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 24 Months · Male only
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2034
U.S. locations
3
States / cities
San Francisco, California • Memphis, Tennessee • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Nov 9, 2025 · Synced May 21, 2026, 7:29 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Anti-thymocyte globulin (rabbit), Busulfan, Fludarabine, Thiotepa, CliniMACS, Donor Lymphocyte Infusion
Drug · Device · Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
2 Months and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 30, 2025 · Synced May 21, 2026, 7:29 PM EDT
Completed Phase 3 Interventional
View directory record Official record
Conditions
Immunologic Deficiency Syndrome, Agammaglobulinemia, Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome, Common Variable Immunodeficiency
Interventions
Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified, Dextrose, 5% in Water
Drug
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years to 75 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2004
U.S. locations
9
States / cities
Birmingham, Alabama • Denver, Colorado • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2009 · Synced May 21, 2026, 7:29 PM EDT
Completed Phase 1 Interventional Results available
View directory record Official record
Conditions
Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
Interventions
Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
Biological · Drug
Lead sponsor
Duke University
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 12, 2014 · Synced May 21, 2026, 7:29 PM EDT
Active, not recruiting Not applicable Interventional
View directory record Official record
Conditions
SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome, Bare Lymphocyte Syndrome, Common Variable Immunodeficiency, Chronic Granulomatous Disease, CD40 Ligand Deficiency, Hyper IgM Syndrome, X-linked Lymphoproliferative Disease, Hemophagocytic Lymphohistiocytosis, Griscelli Syndrome, Chediak-Higashi Syndrome, Langerhan's Cell Histiocytosis
Interventions
Alemtuzumab 0.3 mg, Cyclophosphamide, Busulfan, Stem Cell Transplantation, Fludarabine phosphate 40 mg, Melphalan, Alemtuzumab 0.2 mg, Fludarabine phosphate 30 mg, MESNA
Drug · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 50 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 14, 2026 · Synced May 21, 2026, 7:29 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional
View directory record Official record
Conditions
X-linked Severe Combined Immunodeficiency, X-SCID, XSCID
Interventions
Plerixafor, Filgrastim, Palifermin, Busulfan, Base-edited hematopoietic stem and progenitor cells
Genetic · Drug · Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 99 Years · Male only
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2034
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 16, 2026 · Synced May 21, 2026, 7:29 PM EDT
Recruiting Phase 2 Interventional
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced May 21, 2026, 7:29 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Gene-Transduced Autologous CD34+ Stem Cells
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Months to 20 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced May 21, 2026, 7:29 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Interventions
Human Placental Derived Stem Cell
Drug
Lead sponsor
New York Medical College
Other
Eligibility
0 Years to 55 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
3
States / cities
Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 24, 2022 · Synced May 21, 2026, 7:29 PM EDT
Withdrawn Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Severe Combined Immunodeficiency Due to ADA Deficiency
Interventions
OTL-101
Biological
Lead sponsor
University of California, Los Angeles
Other
Eligibility
30 Days to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 2, 2022 · Synced May 21, 2026, 7:29 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Adenosine Deaminase Deficiency, ADA-SCID
Interventions
Lentiviral Gene Transfer
Genetic
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Year to 65 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 26, 2017 · Synced May 21, 2026, 7:29 PM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Severe Aplastic Anemia (SAA)
Interventions
REGN7257
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
2
States / cities
Cleveland, Ohio • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced May 21, 2026, 7:29 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Gene transfer
Biological
Lead sponsor
David Williams
Other
Eligibility
Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2023
U.S. locations
3
States / cities
Los Angeles, California • Boston, Massachusetts • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 20, 2026 · Synced May 21, 2026, 7:29 PM EDT
Enrolling by invitation No phase listed Observational
View directory record Official record
Conditions
Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID)
Interventions
autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
Biological
Lead sponsor
University of California, Los Angeles
Other
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2040
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced May 21, 2026, 7:29 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Severe Combined Immunodeficiency, Malignancy, Hematologic, Neuroblastoma, Neoplasm, Mucopolysaccharidosis I
Interventions
Venipuncture
Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2024
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 29, 2024 · Synced May 21, 2026, 7:29 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
13 Years to 17 Years · Female only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced May 21, 2026, 7:29 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
6 Months to 99 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced May 21, 2026, 7:29 PM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Transplant Conditioning with Mobilization Only, Transplant Conditioning with Mobilization and Alemtuzumab
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 3 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 30, 2018 · Synced May 21, 2026, 7:29 PM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Severe Combined Immunodeficiency, X Linked, Gene Therapy
Interventions
autologous CD34+ cell transduced with G2SCID vector
Biological
Lead sponsor
David Williams
Other
Eligibility
0 Years to 5 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
4
States / cities
Los Angeles, California • Atlanta, Georgia • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2025 · Synced May 21, 2026, 7:29 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency
Interventions
EZN-2279, Adagen
Biological
Lead sponsor
Leadiant Biosciences, Inc.
Industry
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
6
States / cities
Los Angeles, California • San Francisco, California • Denver, Colorado + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 15, 2020 · Synced May 21, 2026, 7:29 PM EDT
Recruiting Phase 2 Interventional
View directory record Official record
Conditions
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome, STAT 1 Gain of Function, STAT 3 Gain of Function, Hypomorphic RAG1 Deficiency, CD40 Ligand Deficiency, Mendelian Susceptibility to Mycobacterial Disease, GATA2 Associated Immunodeficiency, CD40 Deficiency, Hypomorphic RAG2 Deficiency, Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome, Omenn Syndrome, Chronic Granulomatous Disease
Interventions
Hematopoietic stem cell transplant (HSCT)
Biological
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 40 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced May 21, 2026, 7:29 PM EDT
Withdrawn Phase 2 Interventional
View directory record Official record
Conditions
Severe Combined Immunodeficiency, Transplacental Maternal Engraftment, Stem Cell Transplant
Interventions
Sirolimus
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
1 Day to 1 Year
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Dec 2, 2015 · Synced May 21, 2026, 7:29 PM EDT