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

NCT06178432 · Status: NOT_YET_RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2023-12-21

No results posted yet for this study

Summary

This is a single-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of CRG003 injection in participants with late onset Pompe disease (LOPD), with a long-term follow-up period of 5 years.

CRG003 (BBM-G102) injection is an adeno-associated virus (AAV) gene therapy product for treating Pompe disease to stably express active GAA enzyme in the liver on a long-term basis after the injection.

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.

Sponsors & Collaborators

  • Huashan Hospital

    lead OTHER

Principal Investigators

  • Chongbo Zhao, MD,PhD · Huashan Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-12-31
Primary Completion
2024-12-31
Completion
2028-12-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06178432 on ClinicalTrials.gov