1 active not recruiting, 1 completed, 1 recruiting. 2 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Active not recruiting 1, Recruiting 1 |
| Phase 2 | 1 | Active not recruiting 1 |
Follow Gaucher Disease, Type 2: new trials by email Literature Review of Gaucher Disease, Type 2 trials Research Report on Gaucher Disease, Type 2
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2 GM1 Gangliosidosis, GM2 Gangliosidosis | Completed | Idorsia Pharmaceuticals Ltd. | 226 | 2019-07-31 | 2021-11-08 |
Gaucher Disease (3) · Gangliosidoses, GM2 (1) · Gangliosidosis, GM1 (1) · Mucopolysaccharidosis IV (1) · Mucopolysaccharidosis VI (1) · Mucopolysaccharidosis VII (1) · Sandhoff Disease (1) · Sudden Infant Death (1) · Tay-Sachs Disease (1) · Tay-Sachs Disease, AB Variant (1) · Wolman Disease (1)
Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Lipid Metabolism Disorders (3) · Lipid Metabolism, Inborn Errors (3) · Lipidoses (3) · Lysosomal Storage Diseases (3) · Lysosomal Storage Diseases, Nervous System (3) · Metabolic Diseases (3) · Metabolism, Inborn Errors (3) · Nervous System Diseases (3) · Nutritional and Metabolic Diseases (3)
GAA protein, human (1) · GALNS protein, human (1) · Iduronidase (1) · Methylprednisolone (1) · Prednisone (1) · Sebelipase alfa (1) · Sirolimus (1) · galsulfase (1) · idursulfase (1) · vestronidase alfa (1)
Idorsia Pharmaceuticals Ltd. (1) · Prevail Therapeutics (1) · University of California, San Francisco (1)
United States (3) · United Kingdom (2) · Belgium (1) · Brazil (1) · France (1) · Germany (1) · Italy (1) · Portugal (1) · Spain (1) · Switzerland (1)
Hamzeh Migdadi, M.D. (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
2019 (1)