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Clinical trial

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

NCT05567627 is registered in ClinicalTrials.gov with Seventh Medical Center of PLA General Hospital as lead sponsor.

NCT NCT05567627
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Registry record

As filed in ClinicalTrials.gov.

Title
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
Lead sponsor
Seventh Medical Center of PLA General Hospital
Phase
Not applicable
Overall status
Unknown status
Start date
August 1, 2022
Completion date
September 30, 2025
Enrollment
6 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

infantile-onset pompe disease