22 completed, 9 terminated, 5 active not recruiting, 3 recruiting. 21 Phase 2, 17 Phase 1, 4 Early Phase 1, 4 Not applicable, 1 Phase 3.
Follow Chronic Granulomatous Disease: new trials by email Literature Review of Chronic Granulomatous Disease trials Research Report on Chronic Granulomatous Disease
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Lentiviral Gene Therapy for CGD Infusion of lentiviral TYF-CGD-modified autologous stem cells - Chronic Granulomatous Disease | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-07-01 | 2026-08-26 |
| Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD) Hematopoietic stem cell transplant (HSCT) - Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder | Phase 2 | Recruiting | Paul Szabolcs | 25 | 2026-05-04 | 2026-05-15 |
| A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD ) PM359 - Chronic Granulomatous Disease, Granulomatous Disease, Chronic | Phase 1 / Phase 2 | Enrolling by invitation | Prime Medicine, Inc. | 12 | 2024-10-17 | 2026-04-06 |
| pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD) Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47 - p47, Autosomal Recessive | Phase 1 / Phase 2 | Enrolling by invitation | National Institute of Allergy and Infectious Diseases (NIAID) | 10 | 2024-06-25 | 2026-09-15 |
| Neutrophil Oxidative Burst in Early and Late Onset Pediatric Inflammatory Bowel Disease Phagoburst, Lymphocytes subsets, Genetic testing - Chronic Granulomatous Disease | Unknown | National Institute for Mother and Child Health Alessandrescu Rusescu | 150 | 2023-11-01 | 2023-12-08 | |
| Natural History of Intestinal Inflammation in People With Primary Immune Dysregulations Chronic Granulomatous Disease, Inflammatory Bowel Disease | Withdrawn | National Institute of Allergy and Infectious Diseases (NIAID) | 2023-09-08 | 2023-09-13 | ||
| Whole Blood Biospecimen Collection for Subjects With Chronic Granulomatous Disease (CGD) Specimen Donation - Chronic Granulomatous Disease | Terminated | Sanguine Biosciences | 6 | 2023-05-23 | 2024-04-18 | |
| Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD) JSP191 - Chronic Granulomatous Disease, CGD | Early Phase 1 | Active not recruiting | National Institute of Allergy and Infectious Diseases (NIAID) | 7 | 2023-01-02 | 2026-08-27 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory Complications XELJANZ (tofacitinib) - Chronic Granulomatous Disease, Inflammatory Gastrointestinal Disease | Phase 1 / Phase 2 | Completed | National Institute of Allergy and Infectious Diseases (NIAID) | 10 | 2022-08-12 | 2026-05-07 |
| NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD) gp91 Grans - Chronic Granulomatous Disease, Infection | Phase 1 | Recruiting | National Institute of Allergy and Infectious Diseases (NIAID) | 25 | 2022-07-22 | 2026-10-02 |
| 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 Sirolimus, Cyclophosphamide, Alemtuzumab - Chronic Granulomatous Disease | Phase 1 / Phase 2 | Recruiting | National Institute of Allergy and Infectious Diseases (NIAID) | 50 | 2022-07-08 | 2026-10-13 |
| Elemental Diet for Treatment of Inflammatory Bowel Disease in Patients With Chronic Granulomatous Disease Neocate Splash - Chronic Granulomatous Disease, Inflammatory Bowel Disease | Phase 2 | Completed | National Institute of Allergy and Infectious Diseases (NIAID) | 3 | 2021-06-23 | 2025-12-11 |
| Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide Busulfan, Alemtuzumab, Cyclophosphamide - Chronic Granulomatous Disease | Early Phase 1 | Active not recruiting | National Institute of Allergy and Infectious Diseases (NIAID) | 4 | 2019-10-28 | 2026-10-16 |
| Plerixafor/G-CSF as Additional Agents for Conditioning Before HSCT in CGD Patients Plerixafor, Gcsf - Chronic Granulomatous Disease | Phase 2 | Unknown | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | 17 | 2019-04-13 | 2019-09-17 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation Thiotepa--single daily dose, Thiotepa--escalated dose - Bone Marrow Failure Syndrome, Thalassemia | Phase 1 / Phase 2 | Completed | University of Florida | 6 | 2018-06-15 | 2023-09-15 |
| 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 Interferon Gamma-1B - Chronic Granulomatous Disease | Completed | University of Colorado, Denver | 9 | 2018-05-16 | 2021-01-22 | |
| Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis - Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission | Phase 2 | Terminated | Roswell Park Cancer Institute | 31 | 2017-12-07 | 2025-07-04 |
| Pioglitazone Therapy for Chronic Granulomatous Disease Pioglitazone - Chronic Granulomatous Disease | Phase 1 / Phase 2 | Terminated | Children's Hospital of Fudan University | 3 | 2017-09-01 | 2019-10-22 |
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Granulomatous Disease, Chronic (47) · Wiskott-Aldrich Syndrome (8) · Lymphohistiocytosis, Hemophagocytic (7) · Chediak-Higashi Syndrome (6) · Immunologic Deficiency Syndromes (6) · Severe Combined Immunodeficiency (6) · Anemia, Diamond-Blackfan (5) · Anemia, Sickle Cell (5) · Common Variable Immunodeficiency (5) · Inflammatory Bowel Diseases (5) · Lymphoproliferative Disorders (5) · Graft vs Host Disease (4) · Hyper-IgM Immunodeficiency Syndrome (4) · Mucopolysaccharidosis I (4) · Cystic Fibrosis (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (47) · Disease Attributes (47) · Genetic Diseases, X-Linked (47) · Hemic and Lymphatic Diseases (47) · Pathologic Processes (47) · Pathological Conditions, Signs and Symptoms (47) · Phagocyte Bactericidal Dysfunction (47) · Chronic Disease (46) · Hematologic Diseases (46) · Leukocyte Disorders (46) · Genetic Diseases, Inborn (45) · Immune System Diseases (45) · Immunologic Deficiency Syndromes (41) · Primary Immunodeficiency Diseases (12) · Digestive System Diseases (11)
Cyclophosphamide (8) · Busulfan (7) · Alemtuzumab (4) · Stem Cell Transplantation (4) · fludarabine phosphate (4) · Sirolimus (3) · Whole-Body Irradiation (3) · fludarabine (3) · Cyclosporine (2) · Interferon-gamma (2) · Melphalan (2) · interferon gamma-1b (2) · Abatacept (1) · Antilymphocyte Serum (1) · Etoposide (1)
National Institute of Allergy and Infectious Diseases (NIAID) (18) · Federal Research Institute of Pediatric Hematology, Oncology and Immunology (2) · University of Colorado, Denver (2) · Baylor College of Medicine (1) · Centre Hospitalier Universitaire de Liege (1) · Children's Hospital Los Angeles (1) · Children's Hospital of Fudan University (1) · Emory University (1) · Fairview University Medical Center (1) · Helixmith Co., Ltd. (1) · Masonic Cancer Center, University of Minnesota (1) · Medical College of Wisconsin (1) · National Cancer Institute (NCI) (1) · National Heart, Lung, and Blood Institute (NHLBI) (1) · National Institute for Mother and Child Health Alessandrescu Rusescu (1)
United States (35) · Canada (2) · China (2) · Russia (2) · South Korea (2) · Belgium (1) · France (1) · Netherlands (1) · Romania (1) · Switzerland (1) · United Kingdom (1)
Elizabeth M Kang, M.D. (7) · Christa S Zerbe, M.D. (3) · Daniel R. Ambruso, MD (2) · Suk S De Ravin, M.D. (2) · Andrew J Lipton, M.D. (1) · Anna Shcherbina, PhD (1) · Biljana Horn, MD (1) · Brian W Pennarola, M.D. (1) · Curt Scharfe, MD, PhD (1) · David M Burger, PharmD PhD (1) · Hyoung Jin Kang, M.D., Ph.D (1) · Janine Reichenbach, PD Dr med (1) · John T Horan, MD (1) · Joong Gon Kim, MD, PhD (1)
PMID 19965657, PMID 21772229, PMID 25459642, PMID 31315600, PMID 31599480, PMID 31745699, PMID 35113934, PMID 35142618, PMID 37042921, PMID 40920799, PMID 41358590
U10HL069294 (1), U24CA076518 (1), UL1TR001082 (1)