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Conditions: IFN-Gamma Therapy, CGD Gene Mutation, CGD Response to IFNg, CGD - Chronic Granulomatous Disease, Immunodeficiency Disease
Interventions: IFN-gamma; 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: 2010 – 2014
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions: Abatacept; Drug
Lead sponsor: Emory University; Other
Eligibility: Up to 21 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Atlanta, Georgia

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: 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
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: 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: p47, Autosomal Recessive, Chronic Granulomatous Disease
Interventions: Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47; Biological
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 3 Years to 65 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

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

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: Bronchogenic Carcinoma, Chronic Granulomatous Disease, Job's Syndrome, Mycobacterium Infection, Wegener's Granulomatosis
Interventions: virtual bronchoscopy; Device
Lead sponsor: National Institutes of Health Clinical Center (CC); NIH
Eligibility: Not listed
Enrollment: 120 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1995 – 2001
U.S. locations: 1
States / cities: Bethesda, Maryland

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: 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

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: 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: Busulfan, Alemtuzumab, Cyclophosphamide, Sirolimus, Total Body Irradiation, Allogeneic peripheral blood stem cell; Drug · Radiation · Biological
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 4 Years to 65 Years
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2034
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Chronic Granulomatous Disease
Interventions: Specimen Donation; Diagnostic Test
Lead sponsor: Sanguine Biosciences; Industry
Eligibility: 18 Years to 85 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: Woburn, Massachusetts

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: Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions: Hematopoietic stem cell transplantation; Procedure
Lead sponsor: Children's Hospital Los Angeles; Other
Eligibility: Up to 21 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2015
U.S. locations: 1
States / cities: Los Angeles, California

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 (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

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: 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

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

Conditions: Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Aplastic Anemia, B-Cell Non-Hodgkin Lymphoma, CD40 Ligand Deficiency, Chronic Granulomatous Disease, Chronic Leukemia in Remission, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Congenital Amegakaryocytic Thrombocytopenia, Congenital Neutropenia, Congenital Pure Red Cell Aplasia, Glanzmann Thrombasthenia, Immunodeficiency Syndrome, Myelodysplastic Syndrome, Myelofibrosis, Myeloproliferative Neoplasm, Paroxysmal Nocturnal Hemoglobinuria, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Non-Hodgkin Lymphoma, Refractory Non-Hodgkin Lymphoma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndrome, Severe Aplastic Anemia, Shwachman-Diamond Syndrome, Sickle Cell Disease, T-Cell Non-Hodgkin Lymphoma, Thalassemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
Interventions: Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Peripheral Blood Stem Cell Transplantation, Total-Body Irradiation; Drug · Other · Procedure + 1 more
Lead sponsor: Roswell Park Cancer Institute; Other
Eligibility: 1 Year to 75 Years
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2023
U.S. locations: 1
States / cities: Buffalo, New York

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Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Posaconazole to Treat Invasive Fungal Infections

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

Gene Therapy for Chronic Granulomatous Disease

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

pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)

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

Skin Immunity Sample Collection Involving Blisters and Biopsies

Diagnostic Effectiveness of Virtual Bronchoscopy

Natural History of Intestinal Inflammation in People With Primary Immune Dysregulations

Radiofrequency Ablation for Liver Abscesses From Chronic Granulomatous Disease

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

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

Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide

Whole Blood Biospecimen Collection for Subjects With Chronic Granulomatous Disease (CGD)

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

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

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

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

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

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

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

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer