RecruitingPhase 1Phase 2NCT06391736

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety, Tolerability, and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Late-onset Pompe Disease

Sponsor

GeneCradle Inc

Enrollment

33 participants

Start Date

Apr 19, 2024

Study Type

INTERVENTIONAL

Conditions

Pompe Disease (Late-onset)

Summary

This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with late-onset Pompe disease （LOPD） who are ≥ 6 years old will be studied.

Eligibility

Min Age: 6 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying GC301 for people with pompe disease (late-onset). The study is currently recruiting participants at 1 location. People eligible for this study include aged 6 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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