1 enrolling by invitation, 1 not yet recruiting, 1 recruiting, 1 terminated. 2 Phase 2, 2 Phase 3, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Recruiting 1, Terminated 1 |
| Phase 3 | 2 | Enrolling by invitation 1, Recruiting 1 |
| Phase 1 | 1 | Terminated 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 |
|---|---|---|---|---|---|---|
| MPS IIIA Disease Monitoring Program No Intervention - MPS IIIA, Sanfilippo Syndrome | Not yet recruiting | Ultragenyx Pharmaceutical Inc | 55 | 2026-11-01 | 2026-10-02 | |
| 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 |
| 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 (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Connective Tissue Diseases (4) · Genetic Diseases, Inborn (4) · Lysosomal Storage Diseases (4) · Metabolic Diseases (4) · Metabolism, Inborn Errors (4) · Mucinoses (4) · Mucopolysaccharidoses (4) · Nutritional and Metabolic Diseases (4) · Skin and Connective Tissue Diseases (4)
Therapeutics (2)
Ultragenyx Pharmaceutical Inc (4)
Australia (3) · Spain (3) · United States (3)
2023 (2), 2015 (1), 2018 (1), 2019 (1)