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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: 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: Chronic Granulomatous Disease, Communicable Disease
Interventions: Gene Therapy Method for CGD, Isolex 300i Magnetic Cell Selector; Drug · Device
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 5 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1995 – 2010
U.S. locations: 1
States / cities: Bethesda, Maryland

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

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 – 2026
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
Interventions: Interferon Gamma-1B; Drug
Lead sponsor: University of Colorado, Denver; Other
Eligibility: 5 Years to 60 Years
Enrollment: 9 participants
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Aurora, Colorado

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, 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
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: 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: 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: 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: 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 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: 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: 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

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

Conditions: Chronic Granulomatous Disease (CGD), X-Linked Chronic Granulomatous Disease
Interventions: Plerixafor, Filgrastim, Palifermin, Busulfan, Base-edited hematopoietic stem and progenitor cells, Sirolimus; Drug · Biological
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 75 Years · Male only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2032
U.S. locations: 1
States / cities: Bethesda, Maryland

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

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, Crohn'S-like IBD, Inflammatory Bowel Disease (IBD)
Interventions: Infliximab; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 10 Years and older
Enrollment: 40 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2006 – 2012
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: 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

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Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

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

Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-up

Effect of IFN-γ on Innate Immune Cells

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

Diagnostic Effectiveness of Virtual Bronchoscopy

Role of Interferon-gamma 1-b (IFN-γ) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease

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

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

Gene Therapy for Chronic Granulomatous Disease

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

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

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

Skin Immunity Sample Collection Involving Blisters and Biopsies

Radiofrequency Ablation for Liver Abscesses From Chronic Granulomatous Disease

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

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

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

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

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

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

Infliximab to Treat Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease

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

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