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

Recruiting Phase 1 Phase 2 Interventional Study
Pompe Disease (Late-onset)
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
6 and older
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
33 (estimated)
Sponsor
GeneCradle Inc · Industry
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About This Trial

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.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Age ≥ 6 years, males or females; * Patient has a diagnosis of LOPD; * Patient has upright FVC ≥ 30% of predicted normal value; * A 6MWT ≥ 40 meters, assistive device allowed; * The patient's legal guardian(s) must be able to understand the purpose and risks of the study and vo…
Contacts

GeneCradle, Inc China

86-13501380583

ind@bj-genecradle.com

CONTACT