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ClinicalTrials.gov public records Last synced May 22, 2026, 2:46 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 419 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Congenital Immunodeficiency Clear all
Completed Not applicable Interventional
View directory record Official record
Conditions
HIV, AIDS, Fetal Alcohol Syndrome, Alcohol Related Neurodevelopmental Disorder
Interventions
Opt-in or Opt-out testing, Focus Group
Behavioral
Lead sponsor
University of Oklahoma
Other
Eligibility
18 Years to 44 Years · Female only
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Oklahoma City, Oklahoma
Source: ClinicalTrials.gov public record
Updated Jan 13, 2016 · Synced May 22, 2026, 2:46 AM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome
Interventions
OTL-103
Genetic
Lead sponsor
Fondazione Telethon
Other
Eligibility
Up to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 28, 2025 · Synced May 22, 2026, 2:46 AM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Hereditary Angioedema (HAE), Angioedema, Bradykinin-mediated Angioedema, C1 Inhibitor Deficiency
Interventions
Deucrictibant XR tablet, Placebo comparator to XR tablet, Deucrictibant 20 mg capsule, Placebo comparator to 20 mg capsule
Drug
Lead sponsor
Institute for Asthma and Allergy
Network
Eligibility
18 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Wheaton, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2025 · Synced May 22, 2026, 2:46 AM EDT
Terminated Phase 3 Interventional Results available
View directory record Official record
Conditions
Ataxia Telangiectasia, Genetic Syndrome
Interventions
EryDex System
Combination Product
Lead sponsor
Quince Therapeutics S.p.A.
Industry
Eligibility
6 Years and older
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Los Angeles, California • Baltimore, Maryland • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 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 Jun 22, 2016 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
9
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 28, 2011 · Synced May 22, 2026, 2:46 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Bloom Syndrome
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
2 Years to 55 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced May 22, 2026, 2:46 AM EDT
Withdrawn Phase 3 Interventional
View directory record Official record
Conditions
Primary Immunodeficiency
Interventions
IGSC 20% daily push versus every 2 weeks pump, IGSC 20% daily push versus once a week pump, IGSC 20% daily push versus 2 times per week pump, IGSC 20% 150 mg/kg
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
2 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
4
States / cities
South Bend, Indiana • Columbus, Ohio • Oklahoma City, Oklahoma + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 9, 2020 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Donidalorsen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
7
States / cities
Scottsdale, Arizona • San Diego, California • Santa Monica, California + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2023 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Aldesleukin
Drug
Lead sponsor
Chiron Corporation
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Toledo, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections, Gastrointestinal Diseases
Interventions
Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
850 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1995
U.S. locations
17
States / cities
San Francisco, California • Denver, Colorado • Wilmington, Delaware + 12 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced May 22, 2026, 2:46 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
rhC1INH
Drug
Lead sponsor
Pharming Technologies B.V.
Industry
Eligibility
13 Years and older
Enrollment
152 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 4, 2024 · Synced May 22, 2026, 2:46 AM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Hypogammaglobulinemia, Congenital Heart Disease
Interventions
IVIG, Placebo
Drug · Other
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Up to 6 Months
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Apr 12, 2017 · Synced May 22, 2026, 2:46 AM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
Viral Infection, Hematopoietic Stem Cell Transplantation (HSCT), Primary Immunodeficiency Disorders (PID)
Interventions
Norovirus -specific T-cell (NST) therapy
Biological
Lead sponsor
Children's National Research Institute
Other
Eligibility
3 Months to 80 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
3
States / cities
Washington D.C., District of Columbia • Baltimore, Maryland • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced May 22, 2026, 2:46 AM EDT
Recruiting Early Phase 1 Interventional
View directory record Official record
Conditions
Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes, Immunodeficiencies, Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome
Interventions
Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
Biological
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 30 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 19, 2026 · Synced May 22, 2026, 2:46 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Primary Immunodeficiency Disease (PIDD)
Interventions
Not listed
Lead sponsor
BriovaRx Infusion Services
Industry
Eligibility
15 Years to 85 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
4
States / cities
Newport Beach, California • Centennial, Colorado • Atlanta, Georgia + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2019 · Synced May 22, 2026, 2:46 AM EDT
Recruiting No phase listed Observational
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 May 22, 2026, 2:46 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Chronic Granulomatous Disease (CGD)
Interventions
Not listed
Lead sponsor
Ensoma
Industry
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
50
States / cities
Montgomery, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 47 more
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Donidalorsen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
6
States / cities
Scottsdale, Arizona • Santa Monica, California • Plymouth, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
HIV Infections
Interventions
Zidovudine
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Day to 3 Months
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
U.S. locations
6
States / cities
Birmingham, Alabama • Stanford, California • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2008 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Primary Immunodeficiency Disease
Interventions
IgG Next Generation (BT595)
Biological
Lead sponsor
Biotest
Industry
Eligibility
2 Years to 75 Years
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
9
States / cities
Birmingham, Alabama • Los Angeles, California • Centennial, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2023 · Synced May 22, 2026, 2:46 AM EDT
Available No phase listed Expanded access
View directory record Official record
Conditions
Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
Interventions
CliniMACS CD34 Reagent System
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
Up to 65 Years
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced May 22, 2026, 2:46 AM EDT
Recruiting Phase 3 Interventional
View directory record Official record
Conditions
Hereditary Angioedema (HAE), Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2, HAE
Interventions
ADX-324 Dose Level 1, ADX-324 Dose Level 2
Drug
Lead sponsor
ADARx Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
15
States / cities
Litchfield Park, Arizona • Little Rock, Arkansas • San Diego, California + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 29, 2026 · Synced May 22, 2026, 2:46 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Candidiasis, Chronic Mucocutaneous
Interventions
Amphotericin B
Drug
Lead sponsor
Matinas BioPharma Nanotechnologies, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 6, 2024 · Synced May 22, 2026, 2:46 AM EDT