3 completed, 2 recruiting, 1 terminated, 1 unknown. 4 Phase 2, 3 Phase 1, 1 Not applicable, 1 Phase 3, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Completed 1, Terminated 1, Unknown 1, Withdrawn 1 |
| Phase 1 | 3 | Completed 1, Recruiting 1, Unknown 1 |
| Not applicable | 1 | Completed 1 |
| Phase 3 | 1 | Withdrawn 1 |
| Phase 4 | 1 | Completed 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 |
|---|---|---|---|---|---|---|
| High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3) Ambroxol - Gaucher Disease, Type 3 | Not applicable | Completed | Agyany Pharma LTD | 12 | 2025-03-01 | 2026-08-24 |
| 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of Gaucher Disease Type 3 Gene therapy, Enzyme Replacement Agent - Gaucher Disease, Type 3 | Phase 2 / Phase 3 | Withdrawn | AVROBIO | 2023-10-01 | 2023-08-09 | |
| MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3) No intervention - Gaucher Disease, Type 3 | Recruiting | University of Minnesota | 5 | 2023-01-01 | 2026-08-04 | |
| 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 |
| PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) Aldurazyme (laronidase) - MPS I, MPS II | Phase 1 | Recruiting | University of California, San Francisco | 10 | 2021-07-01 | 2026-03-17 |
| A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease Elelyso - Gaucher Disease, Type 3 | Phase 4 | Completed | Ari Zimran | 14 | 2020-11-20 | 2024-12-17 |
| 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 |
| Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease velaglucerase alfa - Gaucher Disease, Type 3 | Phase 1 / Phase 2 | Completed | Shire | 7 | 2012-09-14 | 2021-06-11 |
Gaucher Disease (8) · Ataxia (1) · Lysosomal Storage Diseases (1) · Mucopolysaccharidosis IV (1) · Mucopolysaccharidosis VI (1) · Mucopolysaccharidosis VII (1) · Myoclonus (1) · Neurologic Manifestations (1) · Sudden Infant Death (1) · Wolman Disease (1)
Brain Diseases (8) · Brain Diseases, Metabolic (8) · Brain Diseases, Metabolic, Inborn (8) · Central Nervous System Diseases (8) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Genetic Diseases, Inborn (8) · Lipid Metabolism Disorders (8) · Lipid Metabolism, Inborn Errors (8) · Lipidoses (8) · Lysosomal Storage Diseases, Nervous System (8) · Metabolic Diseases (8) · Metabolism, Inborn Errors (8) · Nervous System Diseases (8) · Nutritional and Metabolic Diseases (8) · Sphingolipidoses (8)
taliglucerase alfa (2) · Ambroxol (1) · Enzyme Replacement Therapy (1) · GAA protein, human (1) · GALNS protein, human (1) · Genetic Therapy (1) · Glucosylceramidase (1) · Iduronidase (1) · Sebelipase alfa (1) · Velaglucerase alfa, human (1) · arimoclomol (1) · galsulfase (1) · idursulfase (1) · imiglucerase (1) · vestronidase alfa (1)
AVROBIO (1) · Agyany Pharma LTD (1) · Ari Zimran (1) · CANbridge (Suzhou) Bio-pharma Co., Ltd. (1) · Shire (1) · University of California, San Francisco (1) · University of Minnesota (1) · ZevraDenmark (1)
India (3) · United States (2) · China (1) · Egypt (1) · Israel (1) · Pakistan (1) · Tunisia (1) · Turkey (Türkiye) (1)
Aabha Nagral, MD (1) · Ari Zimran, Prof. (1) · Bing Han, MD (1) · Huma Arshad Cheema, Prof. (1) · Milena Veselinovic, MD, PhD (1) · Tippi MacKenzie, MD (1)
PMID 15324318, PMID 19948615, PMID 21637107, PMID 23510062, PMID 28374935, PMID 28657663, PMID 30337566, PMID 32102934, PMID 37955580, PMID 39891377, PMID 9918480