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Showing 1–24 of 73 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Severe Combined Immunodeficiency Clear all
Completed Phase 3 Interventional

Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients

NCT00220766
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 Sep 29, 2026, 5:12 PM EDT
Completed Phase 3 Interventional Results available

EZN-2279 in Patients With ADA-SCID

NCT01420627
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 Sep 29, 2026, 5:12 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus

NCT04370795
View directory record Official record
Conditions
Severe Combined Immune Deficiency (SCID)
Interventions
Sirolimus, Busulfan, Horse -Anti-thymocyte, G-CSF, Total Body Irradiation (TBI)
Drug · Radiation
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 40 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 30, 2025 · Synced Sep 29, 2026, 5:12 PM EDT
Not listed Phase 1Phase 2 Interventional

Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency

NCT03315078
View directory record Official record
Conditions
X-Linked Combined Immunodeficiency Diseases
Interventions
CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT, Palifermin, Busulfan
Biological · Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
2 Years to 40 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 3, 2019 · Synced Sep 29, 2026, 5:12 PM EDT
No Longer Available No phase listed Expanded access

cliniMACs HUD for T Cell Depletion

NCT02915406
View directory record Official record
Conditions
x Linked Combined Immunodeficiency
Interventions
CliniMACs
Device
Lead sponsor
University of Miami
Other
Eligibility
Up to 65 Years
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Aug 15, 2017 · Synced Sep 29, 2026, 5:12 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 Sep 29, 2026, 5:12 PM EDT
Completed Phase 1 Interventional

Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant

NCT00008450
View directory record Official record
Conditions
Adenosine Deaminase Deficiency, Autosomal Recessive Disorder, Immune System Disorder, Purine-Nucleoside Phosphorylase Deficiency, Severe Combined Immunodeficiency, Severe Combined Immunodeficiency With Absence of T and B Cells, X-Linked Severe Combined Immunodeficiency
Interventions
Allogeneic Bone Marrow Transplantation, Cyclosporine, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Total-Body Irradiation
Procedure · Drug · Other + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2018
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jul 28, 2019 · Synced Sep 29, 2026, 5:12 PM EDT
Completed Phase 2 Interventional

Phase II Trial of Sequential Chemotherapy and Radiotherapy for AIDS-Related Primary Central Nervous System Lymphoma

NCT00000801
View directory record Official record
Conditions
Lymphoma, Non-Hodgkin, HIV Infections
Interventions
Filgrastim, Vincristine sulfate, Doxorubicin hydrochloride, Cyclophosphamide, Cytarabine, Dexamethasone
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
16 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1998
U.S. locations
7
States / cities
San Francisco, California • Denver, Colorado • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 31, 2012 · Synced Sep 29, 2026, 5:12 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 Sep 29, 2026, 5:12 PM EDT
Completed Phase 3 Interventional

Trimetrexate Plus Leucovorin Calcium Rescue Versus Sulfamethoxazole-Trimethoprim in the Treatment of Pneumocystis Carinii Pneumonia (PCP) in Patients With AIDS

NCT00001014
View directory record Official record
Conditions
Pneumonia, Pneumocystis Carinii, HIV Infections
Interventions
Trimetrexate glucuronate, Sulfamethoxazole-Trimethoprim, Leucovorin calcium
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
12 Years and older
Enrollment
302 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1991
U.S. locations
6
States / cities
San Francisco, California • Chicago, Illinois • New Orleans, Louisiana + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced Sep 29, 2026, 5:12 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector

NCT01129544
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 Sep 29, 2026, 5:12 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy

NCT06851767
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 Sep 22, 2026 · Synced Sep 29, 2026, 5:12 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

NCT05432310
View directory record Official record
Conditions
Adenosine Deaminase Severe Combined Immune Deficiency
Interventions
A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme
Combination Product
Lead sponsor
University of California, Los Angeles
Other
Eligibility
1 Month and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Sep 29, 2026, 5:12 PM EDT
Completed Phase 2 Interventional Results available

MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID

NCT00794508
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
ADA gene transfer
Biological
Lead sponsor
Donald B. Kohn, M.D.
Other
Eligibility
1 Month to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 22, 2021 · Synced Sep 29, 2026, 5:12 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 Sep 29, 2026, 5:12 PM EDT
Not listed No phase listed Observational

Molecular and Clinical Studies of Primary Immunodeficiency Diseases

NCT00006319
View directory record Official record
Conditions
Wiskott- Aldrich Syndrome, ADA Deficient SCID
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
3 Years and older
Enrollment
266 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced Sep 29, 2026, 5:12 PM EDT
Suspended Phase 1Phase 2 Interventional

Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants

NCT01512888
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 Sep 29, 2026, 5:12 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 Sep 29, 2026, 5:12 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 Sep 29, 2026, 5:12 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 Sep 29, 2026, 5:12 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 Sep 29, 2026, 5:12 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 Sep 29, 2026, 5:12 PM EDT
Completed Phase 1 Interventional

Granulocyte-Macrophage Colony-Stimulating Factor and Zidovudine: A Phase I Study of Concurrent Administration in Patients With AIDS and Severe ARC

NCT00000711
View directory record Official record
Conditions
HIV Infections, Cytopenias
Interventions
Zidovudine, Sargramostim
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
12 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1990
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced Sep 29, 2026, 5:12 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene

NCT01852071
View directory record Official record
Conditions
ADA-SCID
Interventions
Infusion of autologous EFS-ADA LV CD34+ (OTL-101), busulfan, PEG-ADA ERT
Genetic · Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
1 Month to 17 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
2
States / cities
Los Angeles, California • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 2, 2022 · Synced Sep 29, 2026, 5:12 PM EDT