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

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

Recruiting

Back to Pompe Disease (Late-onset) trials

Phase 1 — Early testing in a small group of people (usually 20–80) to evaluate safety, determine safe dosage, and identify side effects.

Trial locations(1 site)

China

Chinese PLA General Hospital, Beijing

Key details

Phase

Early testing / Safety & dosing

Sponsor

GeneCradle Inc

Enrollment target

~33 participants

Started

April 2024

Primary completion

December 2026

Age range

6 Years and older

Last updated July 2025

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