Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
ConditionPompe Disease (Late-onset)
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age6+
SponsorGeneCradle Inc
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.
Eligibility criteria
Qualifiers
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;
Disqualifiers
Patient who has any history or concurrent clinical organic disease, including cardiovascular and liver diseases, respiratory system, nervous system disease, or any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.
Patient who requires invasive mechanical ventilation, or rely on noninvasive non-non-invasive assisted ventilation when sitting upright;
Patient who is positive for human immunodeficiency (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;
Patient with a history of glucocorticoid allergy;
Trial design
Treatments tested in this trial
- GC301
Treatment groups
33 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov