15 completed, 5 active not recruiting, 4 not yet recruiting, 4 unknown. 34 Phase 2, 12 Phase 1, 4 Phase 3.
Follow Gaucher Disease: new trials by email Literature Review of Gaucher Disease trials Research Report on Gaucher 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 |
|---|---|---|---|---|---|---|
| Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3) AZ-3102 - Gaucher Disease Type 3 | Phase 2 | Not yet recruiting | Azafaros B.V. | 20 | 2027-01-25 | 2026-11-12 |
| 4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma GD2, PSMA and CD276 CAR-T cells - Neuroblastoma | Phase 1 / Phase 2 | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 100 | 2026-12-31 | 2026-10-30 |
| Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders AZ-3102 - Niemann-Pick Disease Type C, GM2 Gangliosidosis | Phase 2 / Phase 3 | Not yet recruiting | Azafaros B.V. | 218 | 2026-12-10 | 2026-10-21 |
| A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease VGN-R08b - Gaucher Disease Type 3 | Phase 1 / Phase 2 | Not yet recruiting | Shanghai Vitalgen BioPharma Co., Ltd. | 12 | 2026-05-14 | 2026-05-22 |
| Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3 Eliglustat Tartrate Capsules - Gaucher Disease | Phase 2 | Unknown | Peking Union Medical College Hospital | 5 | 2024-08-01 | 2024-07-26 |
| A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease LY-M001 - Gaucher Disease Type 1 | Phase 1 / Phase 2 | Recruiting | Lingyi Biotech Co., Ltd. | 12 | 2024-07-05 | 2026-01-26 |
| Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2) FLT201 - Gaucher Disease, Type 1 | Phase 1 / Phase 2 | Active not recruiting | Spur Therapeutics | 75 | 2024-05-13 | 2025-11-04 |
| An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3 Gene therapy, Enzyme Replacement Agent - Gaucher Disease, Type 3 | Phase 2 / Phase 3 | Withdrawn | AVROBIO | 2023-10-01 | 2023-08-09 | |
| A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) LY3884961 - Gaucher Disease, Gaucher Disease, Type 1 | Phase 1 / Phase 2 | Active not recruiting | Prevail Therapeutics | 8 | 2022-12-20 | 2026-08-17 |
| Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease Low-dose CAN103, High-dose CAN103 - Gaucher Disease, Type 1, Gaucher Disease, Type 3 | Phase 1 / Phase 2 | Unknown | CANbridge (Suzhou) Bio-pharma Co., Ltd. | 40 | 2022-07-11 | 2022-07-27 |
| A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy. Enzyme replacement therapy for Gaucher disease in combination with altus care application - Gaucher Disease Type 1 | Phase 2 | Completed | Hadassah Medical Organization | 5 | 2022-04-01 | 2023-09-22 |
| Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) LY3884961, Methylprednisolone, Sirolimus - Gaucher Disease, Type 2 | Phase 1 / Phase 2 | Active not recruiting | Prevail Therapeutics | 7 | 2021-06-29 | 2026-10-22 |
| Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL) LY3884961, Methylprednisolone, Sirolimus - Parkinson Disease | Phase 1 / Phase 2 | Active not recruiting | Prevail Therapeutics | 32 | 2020-01-03 | 2026-10-13 |
| Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease AVR-RD-02 - Gaucher Disease | Phase 1 / Phase 2 | Terminated | AVROBIO | 8 | 2019-05-30 | 2024-01-18 |
| Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy Ambroxol - Gaucher Disease, Type 1 | Phase 2 | Completed | Shaare Zedek Medical Center | 40 | 2019-03-01 | 2023-02-14 |
| Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3 Arimoclomol, Placebo oral capsule - Gaucher Disease, Type 1, Gaucher Disease, Type 3 | Phase 2 | Terminated | ZevraDenmark | 39 | 2018-06-06 | 2023-01-11 |
| Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension venglustat (GZ402671), imiglucerase - Gaucher Disease Type 1, Gaucher Disease Type 3 | Phase 2 | Active not recruiting | Genzyme, a Sanofi Company | 12 | 2017-01-04 | 2026-07-10 |
| Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1) N-acetylcysteine - Gaucher Disease Type 1 | Phase 2 | Completed | University of Minnesota | 33 | 2015-09-01 | 2026-02-18 |
| An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112 PRX-112 - Gaucher Disease | Phase 2 | Completed | Protalix | 10 | 2014-04-01 | 2016-09-28 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Phase 2 | Recruiting | Paul Szabolcs | 100 | 2014-02-04 | 2025-12-15 |
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Adrenoleukodystrophy (2) · Gangliosidosis, GM1 (2) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Metachromatic (2) · Metabolism, Inborn Errors (2) · Rare Diseases (2) · Sphingolipidoses (2) · Anemia (1) · Anemia, Diamond-Blackfan (1) · Anemia, Sickle Cell (1) · Arthritis, Juvenile (1) · Autoimmune Lymphoproliferative Syndrome (1) · Bone Diseases (1) · Bone Diseases, Metabolic (1)
Brain Diseases (34) · Brain Diseases, Metabolic (34) · Brain Diseases, Metabolic, Inborn (34) · Central Nervous System Diseases (34) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (34) · Genetic Diseases, Inborn (34) · Lipid Metabolism Disorders (34) · Lipid Metabolism, Inborn Errors (34) · Lipidoses (34) · Lysosomal Storage Diseases (34) · Lysosomal Storage Diseases, Nervous System (34) · Metabolic Diseases (34) · Nervous System Diseases (34) · Nutritional and Metabolic Diseases (34) · Metabolism, Inborn Errors (32)
Glucosylceramidase (4) · AT2101 (3) · Ambroxol (2) · Enzyme Replacement Therapy (2) · Methylprednisolone (2) · Sirolimus (2) · eliglustat (2) · imiglucerase (2) · Acetylcysteine (1) · Alemtuzumab (1) · Alendronate (1) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Calcium Carbonate (1)
Amicus Therapeutics (3) · Prevail Therapeutics (3) · Shire (3) · AVROBIO (2) · Azafaros B.V. (2) · Genzyme, a Sanofi Company (2) · National Institute of Neurological Disorders and Stroke (NINDS) (2) · CANbridge (Suzhou) Bio-pharma Co., Ltd. (1) · Children's Hospital Medical Center, Cincinnati (1) · Exsar Corporation (1) · Hadassah Medical Organization (1) · Lingyi Biotech Co., Ltd. (1) · Masonic Cancer Center, University of Minnesota (1) · National Center for Research Resources (NCRR) (1) · Paul Szabolcs (1)
United States (19) · Israel (9) · United Kingdom (7) · China (4) · India (3) · Spain (3) · Germany (2) · Argentina (1) · Australia (1) · Brazil (1) · Canada (1) · Egypt (1) · France (1) · Italy (1) · Japan (1)
Aabha Nagral, MD (1) · Aaron Tward, MD, PhD (1) · Ari Zimran (1) · Bing Han (1) · Bing Han, MD (1) · Fengkui Zhang, PhD (1) · Hamzeh Migdadi, M.D. (1) · John Barranger (1) · Milena Veselinovic, MD (1)
PMID 20299511, PMID 20439622, PMID 20713962, PMID 21586746, PMID 23339116, PMID 23860343, PMID 24816856, PMID 24835462, PMID 27866808, PMID 32634238, PMID 32750127, PMID 36739645, PMID 37624739, PMID 38802634
2005 (1), 2014 (1), 2023 (1), U54NS065768 (1)