2 enrolling by invitation. 2 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Enrolling by invitation 2 |
| Phase 2 | 1 | Enrolling by invitation 1 |
Follow Lysosomal Storage Diseases: new trials by email Literature Review of Lysosomal Storage Diseases trials Research Report on Lysosomal Storage Diseases
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 scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU) scAAV9/AGA - Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency | Phase 1 / Phase 2 | Enrolling by invitation | Rare Trait Hope | 9 | 2026-11-01 | 2026-10-27 |
| 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 |
Aspartylglucosamidase (AGA) deficiency (1) · Aspartylglucosaminuria (1) · Mucolipidoses (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Nutritional and Metabolic Diseases (2) · 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) · Lysosomal Storage Diseases, Nervous System (1) · Musculoskeletal Diseases (1)
Rare Trait Hope (1) · The Children's Hospital of Zhejiang University School of Medicine (1)
China (1) · United States (1)