4 not yet recruiting, 1 active not recruiting, 1 enrolling by invitation, 1 withdrawn. 4 Phase 2, 2 Phase 3, 1 Phase 1, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Not yet recruiting 3, Active not recruiting 1 |
| Phase 3 | 2 | Not yet recruiting 2 |
| Phase 1 | 1 | Not yet recruiting 1 |
| Phase 4 | 1 | Withdrawn 1 |
Follow Gaucher Disease Type 3: new trials by email Literature Review of Gaucher Disease Type 3 trials Research Report on Gaucher Disease Type 3
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 | 2026-09-28 | 2026-07-20 |
| Study of Velaglucerase Beta ERT in Children With Gaucher Disease Velaglucerase beta - Gaucher Disease, Gaucher Disease Type 1 | Phase 3 | Not yet recruiting | CANbridge Life Sciences Ltd. | 10 | 2026-09-16 | 2026-09-29 |
| 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 |
| Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3 echo - Gaucher Disease Type 3 | Enrolling by invitation | Noha Osman | 44 | 2024-04-01 | 2024-10-04 | |
| 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 |
| The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease Velaglucerase alfa - Gaucher Disease Type 1, Gaucher Disease Type 3 | Phase 4 | Withdrawn | Baylor Research Institute | 2015-07-01 | 2018-03-02 |
Gaucher Disease (7) · Gangliosidoses, GM2 (1) · Gangliosidosis, GM1 (1) · Niemann-Pick Disease, Type C (1)
Brain Diseases (7) · Brain Diseases, Metabolic (7) · Brain Diseases, Metabolic, Inborn (7) · Central Nervous System Diseases (7) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Genetic Diseases, Inborn (7) · Lipid Metabolism Disorders (7) · Lipid Metabolism, Inborn Errors (7) · Lipidoses (7) · Lysosomal Storage Diseases (7) · Lysosomal Storage Diseases, Nervous System (7) · Metabolic Diseases (7) · Metabolism, Inborn Errors (7) · Nervous System Diseases (7) · Nutritional and Metabolic Diseases (7)
Caves (1) · Glucosylceramidase (1) · imiglucerase (1) · venglustat (1)
Azafaros B.V. (2) · Baylor Research Institute (1) · CANbridge Life Sciences Ltd. (1) · Genzyme, a Sanofi Company (1) · Noha Osman (1) · Shanghai Vitalgen BioPharma Co., Ltd. (1)
United States (2) · China (1) · Egypt (1) · Germany (1) · India (1) · Japan (1) · Pakistan (1) · Turkey (Türkiye) (1) · United Kingdom (1)
Raphael Schiffmann, M.D.,M.H.Sc. (1)
2014 (1), 2023 (1)