Pompe Disease Infantile-Onset Clinical Trial
Official title:
A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Infantile-onset Pompe Disease
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied.
n/a
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT05619900 -
Registry of Patients Diagnosed With Lysosomal Storage Diseases
|
||
| Recruiting |
NCT05083806 -
MSOT in Pompe Disease
|
N/A | |
| Recruiting |
NCT04755751 -
Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease.
|
||
| Recruiting |
NCT04532047 -
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases
|
Phase 1 | |
| Completed |
NCT00763932 -
Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies
|
Phase 2 | |
| Available |
NCT04327973 -
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
|