256. Muscle glycogenosis Clinical trials / Disease details
Clinical trials : 180 / Drugs : 133 - (DrugBank : 29) / Drug target genes : 25 - Drug target pathways : 105
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05567627 (ClinicalTrials.gov) | August 1, 2022 | 25/9/2022 | Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease | Single Arm, Multicenter, Open and Dose-escalation Clinical Study on Safety, Tolerance, and Efficacy of GC301, an AAV-Delivered Gene Transfer Therapy in Patients With Infantile-onset Pompe Disease | Infantile-onset Pompe Disease | Biological: Genetic: GC301 | Seventh Medical Center of PLA General Hospital | GeneCradle Therapeutics, Inc | Recruiting | N/A | 6 Months | All | 6 | N/A | China |