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ClinicalTrials.gov public records Last synced Oct 24, 2026, 2:53 PM EDT

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Showing 385–408 of 425 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Immunodeficiency Syndrome Clear all
Terminated Phase 3 Interventional Results available

Open-Label Extension of EryDex Study IEDAT-04-2022

NCT06664853
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
Dexamethasone sodium phosphate
Drug
Lead sponsor
Quince Therapeutics S.p.A.
Industry
Eligibility
6 Years and older
Enrollment
101 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
5
States / cities
Los Angeles, California • Baltimore, Maryland • New Brunswick, New Jersey + 2 more
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 24, 2026, 2:53 PM EDT
Withdrawn Not applicable Interventional

Effects of Growth Hormone in Chronically Ill Children

NCT00286689
View directory record Official record
Conditions
Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis, Crohn Disease, HIV Infections
Interventions
Growth Hormone, Whole body Protein turnover, DEXA scan
Drug · Procedure
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
3 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 14, 2018 · Synced Oct 24, 2026, 2:53 PM EDT
Completed No phase listed Observational

Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study

NCT00001255
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
ADA PBSC, ADA Umbilical Cord Blood Cells, Transduced Lymphocytes
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1990 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 24, 2026, 2:53 PM EDT
Completed No phase listed Observational

Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)

NCT03399461
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome
Interventions
Patient Interview guide, Caregiver interview guide, Sociodemographic questionnaire, Pediatric quality of life (PedsQL) questionnaire, Clinical questionnaire
Other
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
12 Years to 30 Years · Male only
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Collegeville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 14, 2019 · Synced Oct 24, 2026, 2:53 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

NCT03301168
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immune Deficiency Disorder, Osteopetrosis, Cytopenia, Hemoglobinopathy in Children, Anemia, Aplastic
Interventions
BPX-501 T cells, Rimiducid
Biological · Drug
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
1 Month to 26 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2034
U.S. locations
10
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 12, 2022 · Synced Oct 24, 2026, 2:53 PM EDT
Completed Phase 1 Interventional

Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-up

NCT00001476
View directory record Official record
Conditions
Chronic Granulomatous Disease, Communicable Disease
Interventions
Gene Therapy Method for CGD, Isolex 300i Magnetic Cell Selector
Drug · Device
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
5 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 24, 2026, 2:53 PM EDT
Terminated Phase 2 Interventional Results available

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

NCT03759678
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
IB1001
Drug
Lead sponsor
IntraBio Inc
Industry
Eligibility
6 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 25, 2026 · Synced Oct 24, 2026, 2:53 PM EDT
Completed No phase listed Observational

Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990

NCT02064933
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Male only
Enrollment
305 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
38
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 33 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2020 · Synced Oct 24, 2026, 2:53 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)

NCT01581593
View directory record Official record
Conditions
Primary Immunodeficiency, Agammaglobulinemia, Hypogammaglobulinemia, Antibody Deficiency
Interventions
Kedrion IVIG 10%
Biological
Lead sponsor
Kedrion S.p.A.
Industry
Eligibility
2 Years to 70 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
12
States / cities
Little Rock, Arkansas • North Palm Beach, Florida • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Oct 24, 2026, 2:53 PM EDT
Completed No phase listed Observational

Newborn Screening for Severe Combined Immunodeficiency (SCID) in a High-Risk Population

NCT00845416
View directory record Official record
Conditions
Severe Combined Immunodeficiency, T Cell Lymphocytopenia
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
1 Day to 30 Days
Enrollment
1,800 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
2
States / cities
Chinle, Arizona • Tuba City, Arizona
Source: ClinicalTrials.gov public record
Updated Jul 16, 2012 · Synced Oct 24, 2026, 2:53 PM EDT
Completed Phase 2 Interventional

Comparison of Two Methods in the Treatment of Cytomegalovirus of the Eyes in Patients With AIDS

NCT00001061
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Sevirumab, Foscarnet sodium, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
167 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1998
U.S. locations
20
States / cities
Birmingham, Alabama • Los Angeles, California • San Jose, California + 13 more
Source: ClinicalTrials.gov public record
Updated Nov 1, 2021 · Synced Oct 24, 2026, 2:53 PM EDT
Active, not recruiting Phase 3 Interventional

Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

NCT06669754
View directory record Official record
Conditions
Hereditary Angioedema (HAE)
Interventions
Deucrictibant, Placebo
Drug
Lead sponsor
Pharvaris Netherlands B.V.
Industry
Eligibility
12 Years and older
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
6
States / cities
Little Rock, Arkansas • San Diego, California • Santa Monica, California + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 13, 2026 · Synced Oct 24, 2026, 2:53 PM EDT
Completed Phase 1 Interventional

A Phase I/II Open-Labelled Trial of Intravitreal Ganciclovir Salvage Therapy for AIDS Patients With Active CMV Retinitis Who Are Intolerant of Systemic Therapy

NCT00000673
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years to 65 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1993
U.S. locations
2
States / cities
Miami, Florida • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 27, 2021 · Synced Oct 24, 2026, 2:53 PM EDT
Active, not recruiting Phase 3 Interventional

A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)

NCT06673056
View directory record Official record
Conditions
Ataxia-Telangiectasia, Ataxia-Telangiectasia (A-T)
Interventions
N-Acetyl-L-Leucine, Placebo
Drug · Other
Lead sponsor
IntraBio Inc
Industry
Eligibility
4 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
3
States / cities
Los Angeles, California • Boston, Massachusetts • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 3, 2025 · Synced Oct 24, 2026, 2:53 PM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Evaluate Bacteriophage as a Useful Immunogen in Patients With Primary Immune Deficiency Diseases (PIDD)

NCT01617122
View directory record Official record
Conditions
Primary Immune Deficiency Diseases
Interventions
Bacteriophage OX174
Biological
Lead sponsor
University of South Florida
Other
Eligibility
2 Years to 85 Years
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1995 – 2015
U.S. locations
1
States / cities
St. Petersburg, Florida
Source: ClinicalTrials.gov public record
Updated Jul 17, 2012 · Synced Oct 24, 2026, 2:53 PM EDT
Active, not recruiting Phase 3 Interventional

Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in Pediatric PID Patients (KIDCARES10)

NCT04944979
View directory record Official record
Conditions
Primary Immunodeficiency Disease
Interventions
Kedrion IVIG 10%
Biological
Lead sponsor
Kedrion S.p.A.
Industry
Eligibility
2 Years to 16 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
8
States / cities
San Francisco, California • Centennial, Colorado • St. Petersburg, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated May 29, 2026 · Synced Oct 24, 2026, 2:53 PM EDT
Completed Phase 1Phase 2 Interventional

Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory Complications

NCT05104723
View directory record Official record
Conditions
Chronic Granulomatous Disease, Inflammatory Gastrointestinal Disease, Inflammatory Skin Disease, Inflammatory Lung Disease
Interventions
XELJANZ (tofacitinib)
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 99 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Oct 24, 2026, 2:53 PM EDT
Completed Phase 3 Interventional

Phase 3 Study of Immune Globulin Intravenous (Human)IVIG-SN™ in Subjects With Primary Immunodeficiency

NCT01406470
View directory record Official record
Conditions
Immunologic Deficiency Syndrome
Interventions
Immune Globulin Intravenous (Human) 5% Liquid, IVIG-SN™
Drug
Lead sponsor
Green Cross Corporation
Industry
Eligibility
2 Years to 70 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
9
States / cities
Birmingham, Alabama • North Palm Beach, Florida • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2014 · Synced Oct 24, 2026, 2:53 PM EDT
Completed Phase 2Phase 3 Interventional Results available

A Long Term Safety Study of BCX7353 in Hereditary Angioedema

NCT03472040
View directory record Official record
Conditions
Hereditary Angioedema, HAE, Prophylaxis
Interventions
BCX7353
Drug
Lead sponsor
BioCryst Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
387 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
42
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Bentonville, Arkansas + 39 more
Source: ClinicalTrials.gov public record
Updated Jun 18, 2023 · Synced Oct 24, 2026, 2:53 PM EDT
Completed Phase 3 Interventional Results available

Gammagard Liquid and rHuPH20 in PID

NCT00814320
View directory record Official record
Conditions
Primary Immunodeficiency Diseases (PID)
Interventions
Recombinant human hyaluronidase (rHuPH20)+ immune globulin intravenous (IGIV)
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
2 Years and older
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
13
States / cities
Cypress, California • Irvine, California • Los Angeles, California + 10 more
Source: ClinicalTrials.gov public record
Updated May 19, 2021 · Synced Oct 24, 2026, 2:53 PM EDT
Completed No phase listed Observational

Influences on Female Adolescents' Decisions Regarding Testing for Carrier Status of XSCID

NCT00006335
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 2, 2017 · Synced Oct 24, 2026, 2:53 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema

NCT05121376
View directory record Official record
Conditions
Hereditary Angioedema, HAE
Interventions
Dose 1 of BMN 331, Dose 2 of BMN 331, Dose 3 of BMN 331, Dose 4 of BMN 331, Dose 5 of BMN 331, Dose 6 of BMN 331, Dose 7 of BMN 331
Genetic
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
13
States / cities
Birmingham, Alabama • Scottsdale, Arizona • San Diego, California + 10 more
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced Oct 24, 2026, 2:53 PM EDT
Completed Phase 1 Interventional

A Study of CDX-1140 (CD40) as Monotherapy or in Combination in Patients With Advanced Malignancies

NCT03329950
View directory record Official record
Conditions
Melanoma, Non-small Cell Lung Cancer, Breast Cancer, Gastric Cancer, Renal Cell Carcinoma, Ovarian Cancer, Cholangiocarcinoma, Bladder Urothelial Carcinoma, Pancreatic Adenocarcinoma, Colorectal Cancer, Esophageal Cancer, Hepatic Cancer, Head and Neck Cancer, Primary Peritoneal Cancer, Fallopian Tube Cancer, Other Solid Tumors, Diffuse Large B-cell Lymphoma (DLBCL), Mantle Cell Lymphoma, Indolent B-cell Lymphomas, Non-Hodgkin Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Waldenstrom's Disease, Marginal Zone Lymphoma, Mucosa Associated Lymphoid Tissue, Small Lymphocytic Leukemia
Interventions
CDX-1140, CDX-301, pembrolizumab, Chemotherapy
Drug
Lead sponsor
Celldex Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
132 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
11
States / cities
Scottsdale, Arizona • Atlanta, Georgia • Augusta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 28, 2024 · Synced Oct 24, 2026, 2:53 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Investigation of Immune Disorders and Deficiencies

NCT01981785
View directory record Official record
Conditions
Primary Immune Deficiencies, Autoimmune Diseases, Inflammatory Diseases, Common Variable Immune Deficiencies, Hypogammaglobulinemia
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
1 Day and older
Enrollment
343 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2018
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 4, 2017 · Synced Oct 24, 2026, 2:53 PM EDT