1 recruiting, 1 terminated. 2 Phase 2, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Recruiting 1, Terminated 1 |
| Phase 1 | 1 | Terminated 1 |
| Phase 3 | 1 | Recruiting 1 |
Follow MPS IIIA: new trials by email Literature Review of MPS IIIA trials Research Report on MPS IIIA
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 |
|---|---|---|---|---|---|---|
| Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease ABO-102 - MPS IIIA, Sanfilippo Syndrome | Phase 1 / Phase 2 | Terminated | Ultragenyx Pharmaceutical Inc | 5 | 2019-09-18 | 2023-07-25 |
| Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy - MPS IIIA, Sanfilippo Syndrome | Phase 2 / Phase 3 | Recruiting | Ultragenyx Pharmaceutical Inc | 36 | 2016-04-25 | 2026-09-22 |
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)
Therapeutics (1)
Ultragenyx Pharmaceutical Inc (2)
Australia (2) · Spain (2) · United States (2)
2015 (1), 2018 (1), 2023 (1)