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Showing 1–24 of 62 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 2 Interventional Results available

Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)

NCT01998633
View directory record Official record
Conditions
Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1, Leukocyte Adhesion Deficiency, IPEX
Interventions
Hematopoietic Stem Cell Transplant
Biological
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
4 Months to 45 Years
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
19
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Atlanta, Georgia + 16 more
Source: ClinicalTrials.gov public record
Updated Dec 7, 2022 · Synced Sep 7, 2026, 4:40 AM EDT
Recruiting Phase 1Phase 2 Interventional

Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists

NCT05463133
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Sirolimus, Cyclophosphamide, Alemtuzumab, Busulfan, Pheripheral blood stem cells, Emapalumab-Izsg, Tociluzumab, Total Body Irradiation
Drug · Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
4 Years to 65 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 13, 2026 · Synced Sep 7, 2026, 4:40 AM EDT
Completed Phase 3 Interventional

Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care

NCT00023192
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
T-Cell Depleted & CD34+Select/w/StemCell Enriched Product
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 7, 2026, 4:40 AM EDT
Completed Phase 2 Interventional

Elemental Diet for Treatment of Inflammatory Bowel Disease in Patients With Chronic Granulomatous Disease

NCT03983837
View directory record Official record
Conditions
Chronic Granulomatous Disease, Inflammatory Bowel Disease
Interventions
Neocate Splash
Other
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
8 Years to 65 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 10, 2025 · Synced Sep 7, 2026, 4:40 AM EDT
Not listed No phase listed Observational

Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995

NCT02082353
View directory record Official record
Conditions
Granulomatous Disease, Chronic
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
1,480 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
39
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 34 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2021 · Synced Sep 7, 2026, 4:40 AM 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 Aug 31, 2026 · Synced Sep 7, 2026, 4:40 AM EDT
Recruiting Phase 2 Interventional

Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)

NCT07284641
View directory record Official record
Conditions
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome, STAT 1 Gain of Function, STAT 3 Gain of Function, Hypomorphic RAG1 Deficiency, CD40 Ligand Deficiency, Mendelian Susceptibility to Mycobacterial Disease, GATA2 Associated Immunodeficiency, CD40 Deficiency, Hypomorphic RAG2 Deficiency, Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome, Omenn Syndrome, Chronic Granulomatous Disease
Interventions
Hematopoietic stem cell transplant (HSCT)
Biological
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 40 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Sep 7, 2026, 4:40 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

Natural History of Intestinal Inflammation in People With Primary Immune Dysregulations

NCT03278912
View directory record Official record
Conditions
Chronic Granulomatous Disease, Inflammatory Bowel Disease
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years and older
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 12, 2023 · Synced Sep 7, 2026, 4:40 AM EDT
Active, not recruiting Phase 2 Interventional

Immune Disorder HSCT Protocol

NCT01821781
View directory record Official record
Conditions
Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Hyper-IgM, DiGeorge Syndrome, Chediak-Higashi Syndrome, Common Variable Immune Deficiency, Immune Dysregulatory Disorders, Hemophagocytic Lymphohistiocytosis, IPEX, Autoimmune Lymphoproliferative Syndrome, X-linked Lymphoproliferative Syndrome
Interventions
Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced Sep 7, 2026, 4:40 AM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced Sep 7, 2026, 4:40 AM EDT
Terminated Not applicable Interventional

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

NCT00006054
View directory record Official record
Conditions
Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Hyper IgM Syndrome, Severe Combined Immunodeficiency, Leukocyte Adhesion Deficiency Syndrome, Virus-Associated Hemophagocytic Syndrome
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, methotrexate, methylprednisolone, prednisone, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
Up to 35 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2002
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 14, 2009 · Synced Sep 7, 2026, 4:40 AM EDT
Completed Phase 1Phase 2 Interventional

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

NCT02234934
View directory record Official record
Conditions
Granulomatous Disease, Chronic, X-linked
Interventions
Lentiviral G1XCGD Gene Therapy
Biological
Lead sponsor
University of California, Los Angeles
Other
Eligibility
23 Months and older · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
3
States / cities
Los Angeles, California • Bethesda, Maryland • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 1, 2025 · Synced Sep 7, 2026, 4:40 AM EDT
Recruiting Phase 1Phase 2 Interventional

An Open-Label, Proof of Consent Study of Vorinostat for the Treatment of Mdoerate-to-Severe Crohn s Disease and Maintenance Therapy With Ustekinumab

NCT03167437
View directory record Official record
Conditions
Crohn's Disease
Interventions
Vorinostat, Ustekinumab
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 65 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 7, 2026, 4:40 AM EDT
Completed No phase listed Observational

Genetic Disorders of Mucociliary Clearance in Nontuberculous Mycobacterial Lung Disease

NCT00368446
View directory record Official record
Conditions
Cystic Fibrosis, Pseudohypoaldosteronism, Nontuberculous Mycobacterial Infection, Chronic Granulomatous Disease, Primary Ciliary Dyskinesia
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 65 Years
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Sep 7, 2026, 4:40 AM 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 Aug 27, 2026 · Synced Sep 7, 2026, 4:40 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

NCT03513328
View directory record Official record
Conditions
Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
Interventions
Thiotepa--single daily dose, Thiotepa--escalated dose
Drug
Lead sponsor
University of Florida
Other
Eligibility
3 Months to 39 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 14, 2023 · Synced Sep 7, 2026, 4:40 AM EDT
Completed No phase listed Observational

Evaluating the Transition From Pediatric to Adult Care Among Adolescents With Chronic Granulomatous Disease

NCT02233036
View directory record Official record
Conditions
CGD
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 26 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 4, 2018 · Synced Sep 7, 2026, 4:40 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease

NCT02629120
View directory record Official record
Conditions
Chronic Granulomatous Disease Transplant
Interventions
Alemtuzumab, Busulfan, Sirolimus, Cyclophosphamide, Total Body Irradiation, Peripheral blood stem cells
Drug · Radiation · Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
4 Years to 65 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Sep 7, 2026, 4:40 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease

NCT06876363
View directory record Official record
Conditions
X-Linked Chronic Granulomatous Disease
Interventions
EN-374
Genetic
Lead sponsor
Ensoma
Industry
Eligibility
3 Months and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
8
States / cities
Los Angeles, California • San Francisco, California • St. Petersburg, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Sep 7, 2026, 4:40 AM 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 19, 2025 · Synced Sep 7, 2026, 4:40 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Effect of IFN-γ on Innate Immune Cells

NCT02609932
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Administration of drug (Interferon-gamma 1-b) subcutaneously
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 60 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2019
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Feb 14, 2019 · Synced Sep 7, 2026, 4:40 AM EDT
Recruiting Phase 1Phase 2 Interventional

Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease

NCT06325709
View directory record Official record
Conditions
Chronic Granulomatous Disease (CGD), X-Linked Chronic Granulomatous Disease
Interventions
Campath, Sirolimus, Base-edited hematopoietic stem and progenitor cells, Busulfan, Palifermin, Filgrastim, Plerixafor
Drug · Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 75 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Sep 7, 2026, 4:40 AM EDT
Completed Phase 1 Interventional

Stem Cell Transplant Following Low-Intensity Chemotherapy to Treat Chronic Granulomatous Disease

NCT00001765
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Nexell Isolex with T-cell Depletion, Baxter isolex 300i
Device
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 7, 2026, 4:40 AM EDT
Recruiting Phase 1 Interventional

NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)

NCT05189925
View directory record Official record
Conditions
Chronic Granulomatous Disease, Infection
Interventions
gp91 Grans
Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 75 Years · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 7, 2026, 4:40 AM EDT