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ClinicalTrials.gov public records Last synced Oct 24, 2026, 10:39 AM EDT

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Showing 361–384 of 425 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Immunodeficiency Syndrome Clear all
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 Sep 28, 2026 · Synced Oct 24, 2026, 10:39 AM EDT
Completed Phase 3 Interventional Results available

Pharmacokinetics (PK) and Safety of Subgam-VF in Primary Immunodeficiency Diseases

NCT01884311
View directory record Official record
Conditions
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinaemia, Hyperimmunoglobulin M Syndrome
Interventions
Subgam
Biological
Lead sponsor
Bio Products Laboratory
Other
Eligibility
2 Years to 75 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
16
States / cities
Chandler, Arizona • Irvine, California • San Diego, California + 12 more
Source: ClinicalTrials.gov public record
Updated Sep 12, 2018 · Synced Oct 24, 2026, 10:39 AM EDT
Completed Phase 1 Interventional

Thymus Transplantation With Immunosuppression

NCT00579709
View directory record Official record
Conditions
DiGeorge Syndrome, DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete DiGeorge Syndrome
Interventions
Thymus Tissue for Transplantation
Biological
Lead sponsor
Sumitomo Pharma Switzerland GmbH
Industry
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 4, 2022 · Synced Oct 24, 2026, 10:39 AM 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 31, 2024 · Synced Oct 24, 2026, 10:39 AM EDT
Completed Phase 3 Interventional Results available

High Infusion Rate Study of Immunoglobulin Intravenous (Human) 10% (NewGam)

NCT01313507
View directory record Official record
Conditions
Primary Immunodeficiency Disease
Interventions
NewGam
Biological
Lead sponsor
Octapharma
Industry
Eligibility
2 Years to 75 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
6
States / cities
Irvine, California • Centennial, Colorado • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 29, 2017 · Synced Oct 24, 2026, 10:39 AM EDT
Completed Phase 2 Interventional Results available

CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

NCT01095510
View directory record Official record
Conditions
Hereditary Angioedema (HAE)
Interventions
CINRYZE
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years to 11 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
8
States / cities
Colorado Springs, Colorado • Tampa, Florida • Chevy Chase, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2021 · Synced Oct 24, 2026, 10:39 AM EDT
Withdrawn Phase 1 Interventional

Radiofrequency Ablation for Liver Abscesses From Chronic Granulomatous Disease

NCT01851460
View directory record Official record
Conditions
Chronic Granulomatous Diseases (CGD) and Liver Lesions
Interventions
Cool-tip RF Ablation System
Device
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 26, 2020 · Synced Oct 24, 2026, 10:39 AM EDT
Recruiting Phase 2 Interventional

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

NCT04232085
View directory record Official record
Conditions
Primary Immune Deficiency Disorder, Immune Deficiency Disease, Bone Marrow Failure, Short Telomere Length, Fanconi Anemia, Non Fanconi DNA-DSB Repair Disorder, Hoyeraal-Hreidarsson Syndrome, Dyskeratosis Congenita, Telomere Biology Disorder, Short Telomere Syndrome
Interventions
Alemtuzumab, Fludarabine, Melphalan, Low Dose Total Body Irradiation, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil
Drug · Radiation
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
4 Months to 50 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 28, 2025 · Synced Oct 24, 2026, 10:39 AM EDT
Completed Phase 3 Interventional Results available

Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects

NCT03277313
View directory record Official record
Conditions
Primary Immunodeficiency Diseases (PID)
Interventions
HYQVIA
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
2 Years to 15 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
19
States / cities
Birmingham, Alabama • Hartford, Connecticut • Miami, Florida + 15 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2023 · Synced Oct 24, 2026, 10:39 AM EDT
Recruiting No phase listed Observational

Detection and Characterization of Infections and Infection Susceptibility

NCT00404560
View directory record Official record
Conditions
Immune Deficiencies
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Month to 100 Years
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 24, 2026, 10:39 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History of Dysregulation and Aging of the Immune System in People With Trisomy 21 With and Without Thymectomy

NCT07416201
View directory record Official record
Conditions
Down Syndrome
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Year to 120 Years
Enrollment
700 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2040
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 19, 2026 · Synced Oct 24, 2026, 10:39 AM EDT
Completed No phase listed Observational

A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II in North America

NCT03845400
View directory record Official record
Conditions
Hereditary Angioedema (HAE)
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Not listed
Enrollment
168 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
23
States / cities
Birmingham, Alabama • Scottsdale, Arizona • San Diego, California + 20 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2023 · Synced Oct 24, 2026, 10:39 AM EDT
Recruiting Not applicable Interventional

Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy

NCT06689800
View directory record Official record
Conditions
Hematologic Malignancy, Solid Tumor, Sickle Cell Disease, Aplastic Anemia, Immune Deficiency, Metabolic Disorder
Interventions
Let's Get REAL
Behavioral
Lead sponsor
Washington University School of Medicine
Other
Eligibility
8 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 24, 2026, 10:39 AM 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 30, 2024 · Synced Oct 24, 2026, 10:39 AM EDT
Completed Phase 1 Interventional

A Study of Ganciclovir in the Treatment of Cytomegalovirus of the Eyes

NCT00001062
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 and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1998
U.S. locations
6
States / cities
Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 24, 2026, 10:39 AM EDT
Completed Phase 2 Interventional

A Phase II Dose-Ranging, Open-Labelled Trial of Foscarnet Salvage Therapy for AIDS Patients With Sight-Threatening CMV Retinitis Who Cannot Be Treated With Ganciclovir Due To Myelosuppression or Treatment Failure

NCT00000691
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years to 65 Years
Enrollment
156 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1992
U.S. locations
11
States / cities
Los Angeles, California • San Francisco, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced Oct 24, 2026, 10:39 AM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1 Esterase Inhibitor for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

NCT02584959
View directory record Official record
Conditions
Hereditary Angioedema (HAE)
Interventions
C1 esterase inhibitor [human] liquid, Placebo
Drug
Lead sponsor
Shire
Industry
Eligibility
12 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
16
States / cities
Scottsdale, Arizona • Santa Monica, California • Walnut Creek, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 24, 2026, 10:39 AM 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 10, 2020 · Synced Oct 24, 2026, 10:39 AM EDT
Terminated Phase 2 Interventional Results available

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders

NCT00358657
View directory record Official record
Conditions
Immunodeficiency Syndrome, Severe Aplastic Anemia, Genetic Disorder
Interventions
Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Sirolimus, Tacrolimus, Total-Body Irradiation
Procedure · Drug · Other + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 55 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2019
U.S. locations
3
States / cities
Nashville, Tennessee • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jan 26, 2021 · Synced Oct 24, 2026, 10:39 AM EDT
Completed No phase listed Observational

Fanconi Syndrome Due to ARVs in HIV-Infected Persons

NCT00499187
View directory record Official record
Conditions
HIV Infections, Fanconi Syndrome, Kidney Disease, Renal Impairment
Interventions
Blood Draws
Procedure
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
10
States / cities
Los Angeles, California • Denver, Colorado • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 17, 2014 · Synced Oct 24, 2026, 10:39 AM EDT
Completed Phase 1 Interventional

A Phase I Pharmacokinetic Study in HIV-Positive Subjects of Oral Ganciclovir and Concomitant Antiretroviral Zidovudine and Didanosine

NCT00002096
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Zidovudine, Didanosine, Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
3
States / cities
Irvine, California • Washington D.C., District of Columbia • Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 24, 2026, 10:39 AM EDT
Completed Phase 2 Interventional Results available

A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE)

NCT03712228
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Factor XIIa antagonist monoclonal antibody, Placebo
Biological · Drug
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years to 65 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
7
States / cities
Orange, California • Walnut Creek, California • Centennial, Colorado + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 8, 2022 · Synced Oct 24, 2026, 10:39 AM 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 Oct 13, 2026 · Synced Oct 24, 2026, 10:39 AM EDT
Completed No phase listed Observational

Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global)

NCT02593188
View directory record Official record
Conditions
Primary Immunodeficiency Diseases (PID)
Interventions
HYQVIA
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
16 Years and older
Enrollment
264 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
30
States / cities
Bakersfield, California • Irvine, California • Redondo Beach, California + 24 more
Source: ClinicalTrials.gov public record
Updated Oct 18, 2022 · Synced Oct 24, 2026, 10:39 AM EDT