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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

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

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

Conditions: Granulomatous Disease, Chronic, Job's Syndrome, Infection
Interventions: Posaconazole; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 2 Years and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2007
U.S. locations: 1
States / cities: Kenilworth, New Jersey

Conditions: Chronic Granulomatous Disease
Interventions: interferon-gamma; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: Not listed
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1992 – 2001
U.S. locations: 1
States / cities: Bethesda, Maryland

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

Conditions: Chronic Granulomatous Disease-associated Colitis
Interventions: MTP 101-LF; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 10 Years to 60 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

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

Conditions: Chronic Granulomatous Disease, Granulomatous Disease, Chronic
Interventions: PM359; Biological
Lead sponsor: Prime Medicine, Inc.; Industry
Eligibility: 6 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2030
U.S. locations: 3
States / cities: Los Angeles, California • Bethesda, Maryland • Nashville, Tennessee

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

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

Conditions: HIV, Cancer, Chronic Granulomatous Disease, Dock 8 Deficiency
Interventions: Not listed
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Enrollment: 272 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2024
U.S. locations: 9
States / cities: Orange, California • Washington D.C., District of Columbia • Tampa, Florida + 6 more

Conditions: Non-Neoplastic Hematologic and Lymphocytic Disorder
Interventions: Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Methotrexate, Mycophenolate Mofetil, Peripheral Blood Stem Cell Transplantation, Tacrolimus, Total-Body Irradiation, Treosulfan, Umbilical Cord Blood Transplantation; Procedure · Biological · Drug + 2 more
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: Up to 49 Years
Enrollment: 98 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2020
U.S. locations: 6
States / cities: Aurora, Colorado • Portland, Oregon • Nashville, Tennessee + 2 more

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

Conditions: Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
Interventions: BPX-501 and Rimiducid; Biological
Lead sponsor: Bellicum Pharmaceuticals; Industry
Eligibility: 4 Months to 55 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: Seattle, Washington

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

Conditions: Chronic Granulomatous Disease
Interventions: Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 3 Years to 55 Years · Male only
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2014
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Atherosclerosis, Chronic Granulomatous Disease (CGD)
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years and older
Enrollment: 156 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2010 – 2020
U.S. locations: 1
States / cities: Bethesda, Maryland

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

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

Conditions: Hyper-Immunoglobulin E. Syndrome (HEIS), Chronic Granulomatous Disease (CGD)
Interventions: Blister induction, Skin Biopsies; Other
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 7 Years to 65 Years
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024
U.S. locations: 1
States / cities: Bethesda, Maryland

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

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

Conditions: Chediak Higashi Syndrome, Chronic Granulomatous Disease, Job's Syndrome, Leukocyte Disorder
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: Not listed
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2005
U.S. locations: 1
States / cities: Bethesda, Maryland

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Natural History of Intestinal Inflammation in People With Primary Immune Dysregulations

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

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

Posaconazole to Treat Invasive Fungal Infections

A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of Childhood

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

Fecal Microbiota Transplantation for Chronic Granulomatous Disease-Associated Colitis

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

A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

Evaluation of Patients With Immune Function Abnormalities

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

Study of Voicing My CHOiCES as a Tool for Advanced Care Planning in Young Adults With Cancer

Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders

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

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

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

Gene Therapy for Chronic Granulomatous Disease

Non-Invasive Assessment of Atherosclerosis in Patients With CGD and Other Disorders of the Immune System

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

Genetic Disorders of Mucociliary Clearance in Nontuberculous Mycobacterial Lung Disease

Skin Immunity Sample Collection Involving Blisters and Biopsies

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

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

Learning and Behavior Problems in Children With Chronic Granulomatous Disease and Related Disorders