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Showing 1–24 of 62 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Severe Combined Immunodeficiency, X Linked Clear all
Completed Phase 2 Interventional Results available

A Phase 2 Safety and Efficacy Study of Tulisokibart (MK-7240/PRA023) in Subjects With Moderately to Severely Active Ulcerative Colitis (MK-7240-005)

NCT04996797
View directory record Official record
Conditions
Ulcerative Colitis
Interventions
Tulisokibart, Companion Diagnostic (CDx) Testing, Placebo
Drug · Device · Other
Lead sponsor
Prometheus Biosciences, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Industry
Eligibility
18 Years and older
Enrollment
178 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
29
States / cities
Mobile, Alabama • Phoenix, Arizona • Sun City, Arizona + 24 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2025 · Synced Jul 21, 2026, 1:27 PM EDT
Enrolling by invitation No phase listed Observational

Natural History Study of SCID Disorders

NCT01186913
View directory record Official record
Conditions
Severe Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome, Reticular Dysgenesis, ADA SCID, XSCID
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
690 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2028
U.S. locations
39
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 34 more
Source: ClinicalTrials.gov public record
Updated Nov 9, 2020 · Synced Jul 21, 2026, 1:27 PM EDT
Completed Phase 1 Interventional

Human Placental-Derived Stem Cell Transplantation

NCT01586455
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 Jul 21, 2026, 1:27 PM EDT
Recruiting Phase 2 Interventional

Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)

NCT07284641
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 Jul 21, 2026, 1:27 PM EDT
Terminated Phase 2 Interventional Results available

Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim

NCT01182675
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 Jul 21, 2026, 1:27 PM EDT
Completed Phase 1 Interventional

Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

NCT00152100
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 Jul 21, 2026, 1:27 PM EDT
Completed Phase 2Phase 3 Interventional

Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases

NCT01019876
View directory record Official record
Conditions
Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency
Interventions
Fludarabine, Cyclophosphamide, Busulfan, Alemtuzumab, Rabbit Anti-thymocyte Globulin, Horse Anti-thymocyte Globulin
Drug
Lead sponsor
Columbia University
Other
Eligibility
Up to 30 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 30, 2024 · Synced Jul 21, 2026, 1:27 PM EDT
Withdrawn Phase 2 Interventional

Sirolimus Prophylaxis for aGVHD in TME SCID

NCT02177760
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 Jul 21, 2026, 1:27 PM EDT
Recruiting Phase 1Phase 2 Interventional

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

NCT03311503
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 Jul 21, 2026, 1:27 PM EDT
Terminated Phase 1 Interventional

REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy

NCT04409080
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 Jun 21, 2026 · Synced Jul 21, 2026, 1:27 PM EDT
Completed No phase listed Observational Results available

Registry Study of Revcovi Treatment in Patients With ADA-SCID

NCT03878069
View directory record Official record
Conditions
Adenosine Deaminase Severe Combined Immunodeficiency
Interventions
elapegademase-lvlr
Biological
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
0 Months to 65 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
14
States / cities
Los Angeles, California • San Diego, California • San Francisco, California + 11 more
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Jul 21, 2026, 1:27 PM EDT
Completed Phase 1 Interventional

Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)

NCT00028236
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 Jul 21, 2026, 1:27 PM EDT
Recruiting Phase 1Phase 2 Interventional

Autologous Gene Therapy for Artemis-Deficient SCID

NCT03538899
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
AProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan
Drug · Device
Lead sponsor
University of California, San Francisco
Other
Eligibility
2 Months and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2038
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Feb 12, 2026 · Synced Jul 21, 2026, 1:27 PM EDT
Enrolling by invitation No phase listed Observational

An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

NCT04049084
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 Jul 21, 2026, 1:27 PM EDT
Completed Phase 1 Interventional

Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency

NCT00018018
View directory record Official record
Conditions
Severe Combined Immunodeficiency Syndrome
Interventions
CD34+ cells transduced with ADA retrovir
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 11, 2019 · Synced Jul 21, 2026, 1:27 PM EDT
Terminated Not applicable Interventional

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

NCT00006054
View directory record Official record
Conditions
Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Hyper IgM Syndrome, Severe Combined Immunodeficiency, Leukocyte Adhesion Deficiency Syndrome, Virus-Associated Hemophagocytic Syndrome
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, methotrexate, methylprednisolone, prednisone, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
Up to 35 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2002
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 14, 2009 · Synced Jul 21, 2026, 1:27 PM EDT
Recruiting No phase listed Observational

Genetic Basis of Immunodeficiency

NCT00055172
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 Jul 16, 2026 · Synced Jul 21, 2026, 1:27 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Evaluation of Patients With Immune Function Abnormalities

NCT00128973
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 Jul 9, 2026 · Synced Jul 21, 2026, 1:27 PM EDT
Active, not recruiting Phase 2 Interventional

Conditioning SCID Infants Diagnosed Early

NCT03619551
View directory record Official record
Conditions
SCID
Interventions
Busulfan, Cell processing for TCRαβ+/CD19+ depletion
Drug · Device
Lead sponsor
Center for International Blood and Marrow Transplant Research
Network
Eligibility
0 Years to 2 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
40
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 35 more
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Jul 21, 2026, 1:27 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

SCID Bu/Flu/ATG Study With T Cell Depletion

NCT02127892
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
unrelated BM with T cell depletion, unrelated cord blood, haplo BM with T cell depletion, unrelated PBSC with T cell depletion
Biological · Device
Lead sponsor
Neena Kapoor, M.D.
Other
Eligibility
Up to 21 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2016
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Sep 17, 2017 · Synced Jul 21, 2026, 1:27 PM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
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 Jul 21, 2026, 1:27 PM EDT
Terminated Phase 2 Interventional Results available

Study of the Safety and Efficacy of Amatuximab in Combination With Pemetrexed and Cisplatin in Subjects With Unresectable Malignant Pleural Mesothelioma (MPM)

NCT02357147
View directory record Official record
Conditions
Mesothelioma, Malignant
Interventions
Placebo, Amatuximab, Pemetrexed, Cisplatin
Drug
Lead sponsor
Morphotek
Industry
Eligibility
18 Years and older
Enrollment
124 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
7
States / cities
La Jolla, California • Newark, Delaware • Bethesda, Maryland + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2020 · Synced Jul 21, 2026, 1:27 PM EDT
Completed No phase listed Observational

Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

NCT00695279
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 Jul 21, 2026, 1:27 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1

NCT00490100
View directory record Official record
Conditions
Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID), Growth Hormone Resistence
Interventions
Increlex
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
2 Years to 20 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2015 · Synced Jul 21, 2026, 1:27 PM EDT