NCT06178432View on ClinicalTrials.gov

Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)

EARLY_PHASE1Not yet recruitingINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

December 31, 2023

Primary Completion Date

December 31, 2024

Study Completion Date

December 31, 2028

Conditions
Pompe Disease (Late-onset)
Interventions
GENETIC

CRG003 injection

The dose of CRG003 will be calculated according to the participant's weight with single intravenous infusion.

Trial Locations (1)

Unknown

Huashan Hospital of Fudan University, Shanghai

All Listed Sponsors
lead

Huashan Hospital

OTHER

NCT06178432 - Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD) | Biotech Hunter | Biotech Hunter