1 active not recruiting, 1 completed. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Active not recruiting 1 |
| Phase 2 | 1 | Active not recruiting 1 |
Follow Mucopolysaccharidosis III-A: new trials by email Literature Review of Mucopolysaccharidosis III-A trials Research Report on Mucopolysaccharidosis III-A
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 |
|---|---|---|---|---|---|---|
| Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA JR-441 - Mucopolysaccharidosis III-A | Phase 1 / Phase 2 | Active not recruiting | JCR Pharmaceuticals Co., Ltd. | 15 | 2023-10-04 | 2026-09-04 |
| A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome) No intervention - Mucopolysaccharidosis III-A | Completed | Sanguine Biosciences | 15 | 2022-09-27 | 2025-01-30 |
Carbohydrate Metabolism, Inborn Errors (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Connective Tissue Diseases (2) · Genetic Diseases, Inborn (2) · Lysosomal Storage Diseases (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Mucinoses (2) · Mucopolysaccharidoses (2) · Nutritional and Metabolic Diseases (2) · Skin and Connective Tissue Diseases (2)
JCR Pharmaceuticals Co., Ltd. (1) · Sanguine Biosciences (1)
Germany (1) · United States (1)