1 enrolling by invitation. 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Enrolling by invitation 1 |
Follow Mucolipidoses: new trials by email Literature Review of Mucolipidoses trials Research Report on Mucolipidoses
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 |
|---|---|---|---|---|---|---|
| Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV AAV9.hMCOLN1co - Mucolipidosis Type IV | Phase 1 | Enrolling by invitation | The Children's Hospital of Zhejiang University School of Medicine | 1 | 2026-01-13 | 2026-02-10 |
Bone Diseases (1) · Bone Diseases, Metabolic (1) · Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1) · Carbohydrate Metabolism, Inborn Errors (1) · Central Nervous System Diseases (1) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Genetic Diseases, Inborn (1) · Lysosomal Storage Diseases (1) · Lysosomal Storage Diseases, Nervous System (1) · Metabolic Diseases (1) · Metabolism, Inborn Errors (1) · Musculoskeletal Diseases (1) · Nervous System Diseases (1)
The Children's Hospital of Zhejiang University School of Medicine (1)
China (1)