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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
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 24, 2026, 5:21 PM EDT
Not listed No phase listed Observational

A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders

NCT00596999
View directory record Official record
Conditions
Hematologic Malignancies
Interventions
UCB and HPDSC
Procedure
Lead sponsor
Celgene Corporation
Industry
Eligibility
Up to 55 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Apr 9, 2012 · Synced Jul 24, 2026, 5:21 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 24, 2026, 5:21 PM EDT
Not listed No phase listed Observational

Patients Treated for SCID (1968-Present)

NCT01346150
View directory record Official record
Conditions
SCID, ADA-SCID, XSCID, Leaky SCID, Omenn Syndrome, Reticular Dysgenesis
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
1,007 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2023
U.S. locations
31
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 26 more
Source: ClinicalTrials.gov public record
Updated Nov 11, 2020 · Synced Jul 24, 2026, 5:21 PM EDT
Completed Phase 1 Interventional Results available

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

NCT00744692
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 Jul 24, 2026, 5:21 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 May 31, 2026 · Synced Jul 24, 2026, 5:21 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 24, 2026, 5:21 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 24, 2026, 5:21 PM EDT
Recruiting No phase listed Observational

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

NCT04528355
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Jul 24, 2026, 5:21 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Establishing Fibroblast-Derived Cell Lines From Skin Biopsies of Patients With Immunodeficiency or Immunodysregulation Disorders

NCT00895271
View directory record Official record
Conditions
Primary Immunodeficiency, DOCK8, Virus Susceptibility
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
2 Years to 85 Years
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 24, 2026, 5:21 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

NCT01852370
View directory record Official record
Conditions
Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified, Severe Chronic Neutropenia, Chronic Granulomatous Disease (CGD), Hyper IgE Syndromes, Hyper IgM Deficiencies, Wiskott-Aldrich Syndrome, Mendelian Susceptibility to Mycobacterial Disease, Common Variable Immune Deficiency (CVID)
Interventions
CD3/CD19 negative allogeneic hematopoietic stem cells
Biological
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 45 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced Jul 24, 2026, 5:21 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)

NCT03597594
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 Jul 24, 2026, 5:21 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 24, 2026, 5:21 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders

NCT00579137
View directory record Official record
Conditions
Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder, Undefined T Cell Deficiency Disorder, Wiskott-Aldrick Syndrome
Interventions
Campath -1H, Fludarabine, Anti-CD45, Stem cell infusion
Biological · Drug · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 1, 2013 · Synced Jul 24, 2026, 5:21 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 24, 2026, 5:21 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 Jul 24, 2026, 5:21 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 24, 2026, 5:21 PM EDT
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 Jul 24, 2026, 5:21 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 24, 2026, 5:21 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 Jul 24, 2026, 5:21 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 Jul 24, 2026, 5:21 PM EDT
Completed No phase listed Observational

Developing Newborn Screening for Infants With Primary Immunodeficiency

NCT00113464
View directory record Official record
Conditions
Immune System Diseases
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jul 24, 2026, 5:21 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 Jul 24, 2026, 5:21 PM EDT
Active, not recruiting Not applicable Interventional

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

NCT01652092
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 Jun 16, 2026 · Synced Jul 24, 2026, 5:21 PM EDT