11 completed, 6 terminated, 4 enrolling by invitation, 4 recruiting. 32 Phase 1, 23 Phase 2.
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Effect of IFN-γ on Innate Immune Cells Administration of drug (Interferon-gamma 1-b) subcutaneously - Chronic Granulomatous Disease | Phase 1 | Completed | University of Colorado, Denver | 20 | 2016-07-01 | 2019-02-15 |
| Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease Lentiviral G1XCGD Gene Therapy - Granulomatous Disease, Chronic, X-linked | Phase 1 / Phase 2 | Completed | University of California, Los Angeles | 10 | 2015-10-29 | 2025-05-02 |
| Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases BPX-501 and Rimiducid - Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis | Phase 1 | Terminated | Bellicum Pharmaceuticals | 1 | 2015-02-01 | 2024-03-25 |
| Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide Sirolimus, Donor peripheral blood stem cells., Cyclophosphamide post transplant - Chronic Granulomatous Disease | Phase 1 / Phase 2 | Terminated | National Institute of Allergy and Infectious Diseases (NIAID) | 7 | 2014-10-23 | 2020-05-12 |
| Bone Marrow Transplant With Abatacept for Non-Malignant Diseases Abatacept - Hurler Syndrome, Fanconi Anemia | Phase 1 | Completed | Emory University | 10 | 2014-01-01 | 2019-12-26 |
| Gene Therapy for X-CGD ex-vivo gene-therapy - X-linked Chronic Granulomatous Disease | Phase 1 / Phase 2 | Unknown | Hubert Serve, Prof., MD | 5 | 2013-07-01 | 2013-08-02 |
| Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD) X vivo gene therapy - X-Linked Chronic Granulomatous Disease | Phase 1 / Phase 2 | Terminated | Genethon | 3 | 2013-06-24 | 2026-04-13 |
| Gene Therapy for X-linked Chronic Granulomatous Disease X vivo gene therapy - X-Linked Chronic Granulomatous Disease | Phase 1 / Phase 2 | Terminated | Genethon | 3 | 2013-06-24 | 2026-04-13 |
| Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases CD3/CD19 negative allogeneic hematopoietic stem cells - Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified | Phase 1 / Phase 2 | Enrolling by invitation | Paul Szabolcs | 16 | 2013-06-20 | 2025-12-15 |
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Anemia, Sickle Cell (4) · Wiskott-Aldrich Syndrome (4) · Anemia, Diamond-Blackfan (3) · Lymphohistiocytosis, Hemophagocytic (3) · Mucopolysaccharidosis I (3) · Severe Combined Immunodeficiency (3) · Thalassemia (3) · Chediak-Higashi Syndrome (2) · Common Variable Immunodeficiency (2) · Dyskeratosis Congenita (2) · Fanconi Anemia (2) · Infections (2) · Inflammatory Bowel Diseases (2) · Job Syndrome (2)
Chronic Disease (32) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (32) · Disease Attributes (32) · Genetic Diseases, X-Linked (32) · Hematologic Diseases (32) · Hemic and Lymphatic Diseases (32) · Immune System Diseases (32) · Immunologic Deficiency Syndromes (32) · Leukocyte Disorders (32) · Pathologic Processes (32) · Pathological Conditions, Signs and Symptoms (32) · Phagocyte Bactericidal Dysfunction (32) · Genetic Diseases, Inborn (31) · Skin and Connective Tissue Diseases (6) · Nutritional and Metabolic Diseases (5)
Busulfan (6) · Sirolimus (4) · Alemtuzumab (2) · Cyclophosphamide (2) · Abatacept (1) · Fibroblast Growth Factor 7 (1) · Filgrastim (1) · Genes (1) · Hematopoietic Stem Cell Transplantation (1) · Infliximab (1) · Interferon-gamma (1) · Pioglitazone (1) · Whole-Body Irradiation (1) · eltrombopag (1) · fludarabine (1)
National Institute of Allergy and Infectious Diseases (NIAID) (12) · Genethon (2) · Bellicum Pharmaceuticals (1) · Children's Hospital Los Angeles (1) · Children's Hospital of Fudan University (1) · Emory University (1) · Ensoma (1) · Great Ormond Street Hospital for Children NHS Foundation Trust (1) · Helixmith Co., Ltd. (1) · Hubert Serve, Prof., MD (1) · Johann Wolfgang Goethe University Hospital (1) · National Institutes of Health Clinical Center (CC) (1) · Paul Szabolcs (1) · Prime Medicine, Inc. (1) · Seoul National University Hospital (1)
United States (22) · United Kingdom (4) · Germany (3) · South Korea (2) · Switzerland (2) · Canada (1) · China (1) · France (1) · Spain (1)
Elizabeth M Kang, M.D. (5) · Suk S De Ravin, M.D. (3) · Adrian Thrasher, MD, PHD (1) · Adrian Thrasher, Professor (1) · Biljana Horn, MD (1) · Caroline Y. Kuo, MD (1) · Christa S Zerbe, M.D. (1) · Daniel R. Ambruso, MD (1) · Dieter Hoelzer, MD, PhD (1) · Donald B. Kohn, MD (1) · Emma Morris, MD, PHD (1) · Hisham Abdel-Azim, MD (1) · Hubert Serve, MD, PHD (1) · Hyoung Jin Kang, M.D., Ph.D (1)
PMID 16582916, PMID 29664709, PMID 31745699, PMID 35113934, PMID 39413163, PMID 41358590
2025 (1), P01HL073104 (1), UL1TR001082 (1)