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

Pompe Disease (Late-onset) Clinical Trial

Official title:
A Single-arm, Open-label, Single-dose Study to Evaluate the Safety, Tolerability, and Efficacy of CRG003 Injection in the Treatment of Late Onset Pompe Disease

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06178432
Study type Interventional
Source Huashan Hospital
Contact Wenhua Zhu, MD,PhD
Phone +8621-52889999
Email zhuwenhua@huashan.org.cn
Status Not yet recruiting
Phase Early Phase 1
Start date December 2023
Completion date December 2028
View Details
See also
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Active, not recruiting NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Phase 1/Phase 2
Completed NCT03285126 - Pompe Gene Therapy- Screening for Eligibility
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Active, not recruiting NCT03694561 - Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
Completed NCT00731081 - Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme® N/A
Completed NCT00455195 - Late-Onset Treatment Study Extension Protocol Phase 4
Recruiting NCT05951790 - Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease N/A
Completed NCT01288027 - Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa Phase 4
Recruiting NCT00567073 - Pompe Pregnancy Sub-Registry
Completed NCT03893240 - Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease N/A
Withdrawn NCT04094948 - Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Phase 2
Completed NCT00250939 - A Study of rhGAA in Patients With Late-Onset Pompe Disease Phase 2
Recruiting NCT03911505 - ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD Phase 3
Recruiting NCT06150820 - A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease N/A
Completed NCT03729362 - A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Phase 3
Completed NCT00268944 - Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support Phase 3
Active, not recruiting NCT04138277 - A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD Phase 3