31 completed, 14 terminated, 11 recruiting, 7 unknown. 37 Phase 2, 32 Phase 1, 6 Not applicable, 5 Early Phase 1, 3 Phase 3.
Follow Granulomatous Disease, Chronic: new trials by email Literature Review of Granulomatous Disease, Chronic trials Research Report on Granulomatous Disease, Chronic
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 |
| Open-label Gene Therapy Study in p47-CGD SGX-001 - Chronic Granulomatous Disease (CGD) | Phase 1 / Phase 2 | Recruiting | Somagenetix AG | 5 | 2026-08-18 | 2026-09-17 |
| 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 |
| Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease Busulfan, Tocilizumab, Eltrombopag - Chronic Granulomatous Disease (CGD) | Phase 1 / Phase 2 | Enrolling by invitation | National Institute of Allergy and Infectious Diseases (NIAID) | 10 | 2025-09-10 | 2026-09-08 |
| Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease EN-374 - X-Linked Chronic Granulomatous Disease | Phase 1 / Phase 2 | Active not recruiting | Ensoma | 15 | 2025-08-05 | 2026-08-21 |
| Skin Immunity Sample Collection Involving Blisters and Biopsies Blister induction, Skin Biopsies - Hyper-Immunoglobulin E. Syndrome (HEIS), Chronic Granulomatous Disease (CGD) | Early Phase 1 | Withdrawn | National Institute of Allergy and Infectious Diseases (NIAID) | 2024-12-09 | 2024-12-13 | |
| Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease Chronic Granulomatous Disease (CGD) | Recruiting | Ensoma | 60 | 2024-11-18 | 2026-08-17 | |
| 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 |
| Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease Campath, Sirolimus, Base-edited hematopoietic stem and progenitor cells - Chronic Granulomatous Disease (CGD), X-Linked Chronic Granulomatous Disease | Phase 1 / Phase 2 | Recruiting | National Institute of Allergy and Infectious Diseases (NIAID) | 15 | 2024-04-17 | 2026-08-13 |
| 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 |
| Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC data collection - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Recruiting | Paul Szabolcs | 50 | 2020-08-20 | 2026-01-13 |
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Wiskott-Aldrich Syndrome (13) · Anemia, Sickle Cell (10) · Chediak-Higashi Syndrome (10) · Lymphohistiocytosis, Hemophagocytic (10) · Severe Combined Immunodeficiency (10) · Anemia, Diamond-Blackfan (9) · Immunologic Deficiency Syndromes (7) · Inflammatory Bowel Diseases (7) · Mucopolysaccharidosis I (7) · Common Variable Immunodeficiency (6) · Hyper-IgM Immunodeficiency Syndrome (6) · Infections (6) · Lymphoproliferative Disorders (6) · Thalassemia (6)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (78) · Disease Attributes (78) · Hemic and Lymphatic Diseases (78) · Pathologic Processes (78) · Pathological Conditions, Signs and Symptoms (78) · Phagocyte Bactericidal Dysfunction (78) · Chronic Disease (77) · Genetic Diseases, X-Linked (77) · Hematologic Diseases (77) · Leukocyte Disorders (77) · Immune System Diseases (76) · Genetic Diseases, Inborn (73) · Immunologic Deficiency Syndromes (71) · Nutritional and Metabolic Diseases (17) · Primary Immunodeficiency Diseases (17)
Busulfan (10) · Cyclophosphamide (8) · Alemtuzumab (6) · Sirolimus (5) · fludarabine (5) · fludarabine phosphate (5) · Stem Cell Transplantation (4) · Cyclosporine (3) · Interferon-gamma (3) · Melphalan (3) · Whole-Body Irradiation (3) · plerixafor (3) · Thiotepa (2) · interferon gamma-1b (2) · posaconazole (2)
National Institute of Allergy and Infectious Diseases (NIAID) (30) · Paul Szabolcs (4) · Children's Hospital Los Angeles (2) · Children's Hospital of Fudan University (2) · Emory University (2) · Ensoma (2) · Federal Research Institute of Pediatric Hematology, Oncology and Immunology (2) · Genethon (2) · University of Colorado, Denver (2) · Baylor College of Medicine (1) · Bellicum Pharmaceuticals (1) · Centre Hospitalier Universitaire de Liege (1) · Fairview University Medical Center (1) · Fred Hutchinson Cancer Center (1) · Great Ormond Street Hospital for Children NHS Foundation Trust (1)
United States (57) · United Kingdom (5) · China (4) · Canada (3) · Germany (3) · France (2) · Russia (2) · South Korea (2) · Switzerland (2) · Belgium (1) · Italy (1) · Netherlands (1) · Romania (1) · Spain (1)
Elizabeth M Kang, M.D. (9) · Paul Szabolcs, MD (4) · Christa S Zerbe, M.D. (3) · Suk S De Ravin, M.D. (3) · Daniel R. Ambruso, MD (2) · Donald B. Kohn, MD (2) · Harry L Malech, M.D. (2) · John I Gallin, M.D. (2) · Adrian Thrasher, MD, PHD (1) · Adrian Thrasher, Professor (1) · Andrew J Lipton, M.D. (1) · Ann Woolfrey (1) · Anna Shcherbina, PhD (1) · Biljana Horn, MD (1)
PMID 16582916, PMID 19965657, PMID 20004776, PMID 20489056, PMID 21772229, PMID 23818196, PMID 24139498, PMID 24290292, PMID 24331379, PMID 25075835, PMID 25239440, PMID 25459642, PMID 26095669, PMID 27262745, PMID 29664709, PMID 31315600, PMID 31376032, PMID 31599480, PMID 31745699, PMID 32634238, PMID 32750127, PMID 35113934, PMID 35142618, PMID 37042921, PMID 37562003, PMID 37659505, PMID 39413163, PMID 40178760, PMID 40920799, PMID 41358590, PMID 42606118
2015 (1), 2025 (1), P01HL073104 (1), U10HL069294 (1), U24CA076518 (1), U54AI082973 (1), UL1TR001082 (1)