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ClinicalTrials.gov public records Last synced Oct 20, 2026, 5:42 PM EDT

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Showing 73–96 of 425 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Immunodeficiency Syndrome Clear all
Recruiting No phase listed Observational

Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform

NCT06999954
View directory record Official record
Conditions
Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia, Heme Malignancy, Shwachman Syndrome, Inherited Bone Marrow Failure, Exocrine Pancreatic Insufficiency, WHIM, ELANE, SBDS Gene Mutation, EFL1 Gene Mutation, DNAJC21 Gene Mutation, SRP54 Gene Mutation, Inherited Cancer Syndrome, Inherited Cancer-Predisposing Syndrome, Neutropenia, Severe Chronic, Neutropenia Other, Neutropenia Chronic Benign, Ribosome Alteration, Ribosomopathy, Immune Deficiency, Inherited BMF Syndrome, Inherited Immunodeficiency Diseases, Cognitive Delay, Mild, Myelodysplastic Syndromes, Pancytopenia
Interventions
Not listed
Lead sponsor
Shwachman-Diamond Syndrome Alliance Inc
Other
Eligibility
Not listed
Enrollment
8,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2088
U.S. locations
1
States / cities
Woburn, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 31, 2025 · Synced Oct 20, 2026, 5:42 PM EDT
Completed Phase 1 Interventional

Use of Busulfan as Conditioning Agent for a Second Stem Cell Transplant

NCT00092937
View directory record Official record
Conditions
Granulomatous Disease, Chronic
Interventions
Busulfan
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 20, 2026, 5:42 PM EDT
Available No phase listed Expanded access

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

NCT01881334
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 20, 2025 · Synced Oct 20, 2026, 5:42 PM EDT
Active, not recruiting Not applicable Interventional Results available

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion

NCT05236764
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia, Hemophagocytic Lymphohistiocytoses, Primary Immunodeficiency Diseases, Hemoglobinopathies, Severe Aplastic Anemia, Cytopenia, Bone Marrow Failure Syndrome, Severe Chronic Active Epstein-Barr Virus Infection
Interventions
CliniMACS
Device
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 20, 2026, 5:42 PM EDT
Completed Not applicable Interventional

Open Label Ganciclovir Therapy for Sight- or Life-Threatening Cytomegalovirus Disease in the Immunocompromised Patient

NCT00002025
View directory record Official record
Conditions
Cytomegalovirus Infections, Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
3 Months and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 20, 2026, 5:42 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

NCT05392114
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Donidalorsen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
12 Years and older
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
16
States / cities
Paradise Valley, Arizona • La Jolla, California • Santa Monica, California + 13 more
Source: ClinicalTrials.gov public record
Updated Dec 12, 2025 · Synced Oct 20, 2026, 5:42 PM EDT
Recruiting No phase listed Observational

Natural History of Noncirrhotic Portal Hypertension

NCT02417740
View directory record Official record
Conditions
Cystic Fibrosis, Immunologic Deficiency Syndrome, Turner Syndrome, Congenital Hepatic Fibrosis, Idiopathic Non-Cirrhotic Portal Hypertension
Interventions
Not listed
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
12 Years to 100 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 20, 2026, 5:42 PM EDT
Completed Not applicable Interventional

The Effects of AZT and Ibuprofen on HIV-Infected Patients With Hemophilia

NCT00002276
View directory record Official record
Conditions
HIV Infections, Hemophilia A
Interventions
Ibuprofen, Zidovudine
Drug
Lead sponsor
University of Pittsburgh
Other
Eligibility
12 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 20, 2026, 5:42 PM EDT
Completed Not applicable Interventional

The Impact of Exercise on Stress, Fatigue, and Quality of Life in Individuals With Primary Immunodeficiency Disease

NCT03211689
View directory record Official record
Conditions
Primary Immune Deficiency Disorder, Common Variable Immunodeficiency, Specific Antibody Deficiency, Hypergammaglobulinemia
Interventions
Exercise Program
Other
Lead sponsor
Stockton University
Other
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Galloway, New Jersey
Source: ClinicalTrials.gov public record
Updated Sep 6, 2018 · Synced Oct 20, 2026, 5:42 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Phase I/II Thymus Transplantation With Immunosuppression #950

NCT00579527
View directory record Official record
Conditions
DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete Atypical DiGeorge Anomaly, Complete DiGeorge Syndrome, Complete Atypical DiGeorge Syndrome
Interventions
Cultured Thymus Tissue for Implantation (CTTI), Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation, Blood Draw, Rabbit anti-thymocyte globulin, Cyclosporine, Tacrolimus, Methylprednisolone or Prednisolone, Daclizumab, Mycophenolate mofetil
Biological · Other · Procedure + 1 more
Lead sponsor
Sumitomo Pharma Switzerland GmbH
Industry
Eligibility
Not listed
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2017
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 25, 2022 · Synced Oct 20, 2026, 5:42 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Tolerability and Safety of IGI, 10% With rHuPH20 in PIDD

NCT01485796
View directory record Official record
Conditions
Primary Immunodeficiency Diseases (PID)
Interventions
Immune Globulin Infusion (Human), 10%, Recombinant human hyaluronidase
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
2 Years and older
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
10
States / cities
Irvine, California • Thornton, Colorado • North Palm Beach, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated May 19, 2021 · Synced Oct 20, 2026, 5:42 PM EDT
Withdrawn Phase 2 Interventional

Sirolimus Prophylaxis for aGVHD in TME SCID

NCT02177760
View directory record Official record
Conditions
Severe Combined Immunodeficiency, Transplacental Maternal Engraftment, Stem Cell Transplant
Interventions
Sirolimus
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
1 Day to 1 Year
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Dec 3, 2015 · Synced Oct 20, 2026, 5:42 PM EDT
Completed Phase 2 Interventional

Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type I

NCT00001905
View directory record Official record
Conditions
Leukocyte Adhesion Deficiency Syndrome
Interventions
Interferon gamma
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 20, 2026, 5:42 PM EDT
Not yet recruiting Early Phase 1 Interventional Accepts healthy volunteers

Haloperidol For Outpatient Symptom Management in Patients Diagnosed With Cannabinoid Hyperemesis Syndrome in the Emergrency Department

NCT07726069
View directory record Official record
Conditions
Cannabinoid Hyperemesis Syndrome
Interventions
Haloperidol
Drug
Lead sponsor
University of Illinois at Chicago
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2028
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 20, 2026, 5:42 PM EDT
Completed No phase listed Observational

The Second Multicenter Hemophilia Cohort Study

NCT00341705
View directory record Official record
Conditions
Liver Decompensation, Hepatocellular Carcinoma, Non-Hodgkin Lymphoma
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
13 Years and older
Enrollment
2,565 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2013
U.S. locations
39
States / cities
Tucson, Arizona • Davis, California • Orange, California + 31 more
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 20, 2026, 5:42 PM EDT
Completed No phase listed Observational

Monitoring of 5% Treatment Naïve Intravenous Immunoglobulin (IVIg) Primary Immunodeficiency Disease (PIDD) Patients Using the CareExchange® System: A Pilot Study Using 5% Gammaplex® IVIg in the Home Setting

NCT02176239
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 7, 2019 · Synced Oct 20, 2026, 5:42 PM EDT
Recruiting Phase 2 Interventional

Adenovirus-specific Cytotoxic T-lymphocytes for Refractory Adenovirus Infection

NCT03266627
View directory record Official record
Conditions
Adenovirus, Primary Immune Deficiency Disorder
Interventions
cytotoxic t-lymphocytes
Drug
Lead sponsor
New York Medical College
Other
Eligibility
1 Month to 79 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
10
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 8, 2025 · Synced Oct 20, 2026, 5:42 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 Oct 16, 2026 · Synced Oct 20, 2026, 5:42 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 29, 2019 · Synced Oct 20, 2026, 5:42 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study of Navenibart (STAR-0215) in Participants With Hereditary Angioedema

NCT05695248
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Navenibart
Drug
Lead sponsor
Astria Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
13
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Bentonville, Arkansas + 10 more
Source: ClinicalTrials.gov public record
Updated May 8, 2026 · Synced Oct 20, 2026, 5:42 PM EDT
Completed Phase 3 Interventional Results available

A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema

NCT01386658
View directory record Official record
Conditions
Hereditary Angioedema (HAE)
Interventions
icatibant
Drug
Lead sponsor
Shire
Industry
Eligibility
2 Years to 17 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
12
States / cities
Los Angeles, California • Tampa, Florida • Shreveport, Louisiana + 9 more
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 20, 2026, 5:42 PM EDT
Completed Phase 2 Interventional Results available

Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease

NCT00578643
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Busulfan, Alemtuzumab, Cyclophosphamide, Fludarabine, Cyclosporine, Stem Cell Infusion
Drug · Biological · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2017
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 9, 2018 · Synced Oct 20, 2026, 5:42 PM EDT
Recruiting Phase 1 Interventional

Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts

NCT04691622
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 26, 2026 · Synced Oct 20, 2026, 5:42 PM EDT
Terminated No phase listed Observational

Pharmacokinetic (PK) and Pharmacodynamics (PD) Study of Ilera Specific Products

NCT03886753
View directory record Official record
Conditions
ALS, Autism Spectrum Disorder, Cancer, Spasticity, Muscle, Dyskinetic Syndrome, Epilepsy, Glaucoma, Huntington Disease, Inflammatory Bowel Disease (IBD), Multiple Sclerosis, Neuropathy, Opioid Use, Parkinson Disease, HIV/AIDS, Ptsd, Intractable Pain, Sickle Cell Disease, Terminal Illness
Interventions
Registry, PK microsampling of blood
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Oct 20, 2026, 5:42 PM EDT