Experimental group:GC301 adeno-associated virus injection

2 trials

Sponsors

The First Medical Center of PLA General Hospital, Children's Hospital, Zhejiang University School of Medicine

Conditions

Infantile-Onset Pompe Disease (IOPD)Pompei disease

Early Phase 1

A single-arm, single-center, open-label clinical study evaluating the safety and efficacy of GC301 adeno-associated virus injection in the treatment of infantile Pompe disease patients

Active, not recruitingChiCTR2600117937

Children's Hospital, Zhejiang University School of MedicineInfantile-Onset Pompe Disease (IOPD)

Start: 2026-02-01Updated: 2026-02-02

Phase 2

A clinical trial of safety, tolerance and preliminary efficacy of GC301 adeno-associated virus injection gene therapy in >1 year old infants with Pompeii disease

Active, not recruitingChiCTR2200065664

The First Medical Center of PLA General HospitalPompei disease

Start: 2023-07-18Updated: 2026-02-09