2 recruiting. 2 Phase 2, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Recruiting 2 |
| Phase 3 | 2 | Recruiting 2 |
Follow Mucinoses: new trials by email Literature Review of Mucinoses trials Research Report on Mucinoses
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 |
|---|---|---|---|---|---|---|
| Study of Cannabidiol in Sanfilippo Syndrome Epidiolex, Placebo - Sanfilippo Syndrome, Mucopolysaccharidosis III | Phase 2 / Phase 3 | Recruiting | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 35 | 2026-04-01 | 2026-09-15 |
| 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) · Mucopolysaccharidoses (2) · Nutritional and Metabolic Diseases (2) · Skin and Connective Tissue Diseases (2)
Cannabidiol (1) · Therapeutics (1)
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (1) · Ultragenyx Pharmaceutical Inc (1)
United States (2) · Australia (1) · Spain (1)
2015 (1), 2023 (1)