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ClinicalTrials.gov public records Last synced Oct 20, 2026, 4:10 PM EDT

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Showing 49–72 of 425 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Immunodeficiency Syndrome Clear all
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 20, 2026, 4:10 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 13, 2026 · Synced Oct 20, 2026, 4:10 PM EDT
Recruiting Phase 1Phase 2 Interventional

Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)

NCT01306019
View directory record Official record
Conditions
X-linked Severe Combined Immunodeficiency (XSCID)
Interventions
Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector, Busulfan, Palifermin
Biological · Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
2 Years to 50 Years · Male only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 19, 2026 · Synced Oct 20, 2026, 4:10 PM EDT
Terminated No phase listed Observational

Host Genetic Factors Influencing HIV1 and HCV Viral Loads and AIDS Clinical Progression in a Hemophilia Cohort (HGDS-3)

NCT00340548
View directory record Official record
Conditions
Hemophilia, AIDS
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
4 Years to 100 Years
Enrollment
333 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2020
U.S. locations
15
States / cities
La Jolla, California • Los Angeles, California • Indianapolis, Indiana + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2020 · Synced Oct 20, 2026, 4:10 PM EDT
Recruiting No phase listed Observational

Study of Chediak-Higashi Syndrome

NCT00005917
View directory record Official record
Conditions
Chediak-Higashi Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 70 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 28, 2026 · Synced Oct 20, 2026, 4:10 PM EDT
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 20, 2026, 4:10 PM EDT
Completed Not applicable Interventional

A Study of the Safety and Tolerance of Long-Term Therapy With Intravenous Cytovene (Ganciclovir Sodium) for Cytomegalovirus Retinitis in Persons With AIDS

NCT00002034
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Zidovudine, Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
8
States / cities
Birmingham, Alabama • Oakland, California • San Francisco, California + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 20, 2026, 4:10 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

NCT01966367
View directory record Official record
Conditions
Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia
Interventions
CD34 Stem Cell Selection Therapy
Biological
Lead sponsor
Diane George
Other
Eligibility
Up to 40 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 14, 2025 · Synced Oct 20, 2026, 4:10 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

NCT07113743
View directory record Official record
Conditions
Chronic Granulomatous Disease (CGD)
Interventions
Busulfan, Tocilizumab, Eltrombopag, Sirolimus, pCCLChimGp91lentiviral vector containing the human gp91 phox (CYBB) gene
Drug · Other
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 60 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 20, 2026, 4:10 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 20, 2026, 4:10 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 20, 2026, 4:10 PM EDT
Completed Phase 1Phase 2 Interventional

Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome

NCT01410825
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome
Interventions
Retrovirus-mediated gene transfer
Biological
Lead sponsor
David Williams
Other
Eligibility
3 Months to 35 Years · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2024
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 14, 2025 · Synced Oct 20, 2026, 4:10 PM EDT
Not listed Not applicable Interventional

Evaluation of Efficacy and Tolerability of Hizentra®

NCT01354587
View directory record Official record
Conditions
Primary Immunodeficiency Disorders
Interventions
Hizentra
Drug
Lead sponsor
University of South Florida
Other
Eligibility
1 Year to 75 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
St. Petersburg, Florida
Source: ClinicalTrials.gov public record
Updated Jul 17, 2012 · Synced Oct 20, 2026, 4:10 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 Oct 13, 2026 · Synced Oct 20, 2026, 4:10 PM EDT
Completed No phase listed Observational

Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995

NCT02116764
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years and older
Enrollment
137 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 20, 2026, 4:10 PM EDT
Recruiting Phase 1Phase 2 Interventional

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

NCT06839456
View directory record Official record
Conditions
Leukemia, High Risk Acute Lymphoblastic Leukemia, High Risk Acute Myeloid Leukemia, Relapse Leukemia, MDS (Myelodysplastic Syndrome), Relapsed Non-Hodgkin Lymphoma, Acquired Aplastic Anemia, Inherited BMF Syndrome, Immunodeficiency, Primary Immune Regulatory Disorder, Hemoglobinopathies, Bone Marrow Failure, Inborn Errors of Metabolism, HLH
Interventions
Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3, Phase 2 Maximum Tolerated Dose determined in Phase 1, Phase 2 Established Dose from prior study, NCT03810196
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
1 Month to 25 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Oct 20, 2026, 4:10 PM EDT
Completed Phase 2 Interventional

The Safety and Effectiveness of Ganciclovir in the Prevention of Cytomegalovirus (CMV) of the Eyes and Disease of the Stomach and Intestines in Patients With HIV

NCT00001034
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 4, 2021 · Synced Oct 20, 2026, 4:10 PM EDT
Completed Phase 3 Interventional Results available

Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases

NCT03939533
View directory record Official record
Conditions
Primary Immune Deficiency Disorder
Interventions
CUTAQUIG
Drug
Lead sponsor
Octapharma
Industry
Eligibility
2 Years to 75 Years
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
18
States / cities
Scottsdale, Arizona • Irvine, California • Santa Barbara, California + 15 more
Source: ClinicalTrials.gov public record
Updated Nov 7, 2023 · Synced Oct 20, 2026, 4:10 PM EDT
Completed Phase 2 Interventional Results available

A Trial of the Pharmacokinetics, Safety, and Tolerability of Subcutaneous Gamunex® in Primary Immunodeficiency

NCT00389324
View directory record Official record
Conditions
Immunologic Deficiency Syndrome
Interventions
Immune Globulin Intravenous (Human)
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
13 Years to 75 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
6
States / cities
Irvine, California • Los Angeles, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 20, 2015 · Synced Oct 20, 2026, 4:10 PM EDT
Not listed No phase listed Observational

Immune Response Following COVID-19 Vaccination

NCT04736524
View directory record Official record
Conditions
IgM and IgG in Blood
Interventions
Rapid antibody test
Other
Lead sponsor
Texas Cardiac Arrhythmia Research Foundation
Other
Eligibility
16 Years to 85 Years
Enrollment
500 participants
Timeline
2021 – 2022
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Feb 3, 2021 · Synced Oct 20, 2026, 4:10 PM 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 20, 2026, 4:10 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 20, 2026, 4:10 PM EDT
Completed Phase 3 Interventional Results available

CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema

NCT05819775
View directory record Official record
Conditions
Hereditary Angioedema (HAE)
Interventions
CSL312
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
2 Years to 11 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
7
States / cities
Litchfield Park, Arizona • Scottsdale, Arizona • Orange, California + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Oct 20, 2026, 4:10 PM EDT
No Longer Available No phase listed Expanded access

Expanded Access for the Prevention of Acute Attacks of 1) Hereditary Angioedema (HAE) in Children and 2) Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH) in Teenagers and Adults

NCT04583007
View directory record Official record
Conditions
Hereditary Angioedema (HAE), Angioedema
Interventions
Lanadelumab 150 mg, Lanadelumab 300 mg
Drug
Lead sponsor
Shire
Industry
Eligibility
2 Years and older
U.S. locations
14
States / cities
Scottsdale, Arizona • Santa Monica, California • Walnut Creek, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 24, 2023 · Synced Oct 20, 2026, 4:10 PM EDT