1 enrolling by invitation, 1 recruiting. 2 Phase 3, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 2 | Enrolling by invitation 1, Recruiting 1 |
| Phase 2 | 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 |
|---|---|---|---|---|---|---|
| Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy - Mucopolysaccharidosis IIIA, MPS IIIA | Phase 3 | Enrolling by invitation | Ultragenyx Pharmaceutical Inc | 41 | 2020-09-28 | 2026-07-17 |
| 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 (2)
Ultragenyx Pharmaceutical Inc (2)
Australia (2) · Spain (2) · United States (2)
2023 (2), 2015 (1), 2019 (1)