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

Plain-language summary

This study evaluates GC301, a gene therapy, in infants with infantile-onset Pompe disease. Infants younger than 6 months who have been diagnosed with the disease may take part. The study is in Phase 1/Phase 2 and is currently active but not recruiting new participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Active, not recruiting

Sponsor

GeneCradle Inc

Intervention

GC301

Start date

6/2/2023

Estimated completion

12/1/2026

Countries

China

Related genes

Locations

• Beijing, China
• Beijing, China
• Changsha, China
• Hangzhou, China
• Zhengzhou, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* Age \< 6 months
* Patient has diagnosis of infantile onset Pompe disease
* The patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed.

Exclusion Criteria:

* Left ventricle ejection fraction (LVEF) \< 40%;
* Patient who has AAV9 neutralizing antibody titer ≥ 1:100；
* Patient who has received enzyme replacement therapy (ERT) more than twice;
* Patient who has respiratory dysfunction before enrollment, including the blood oxygen (O2) saturation level \< 90%, or the partial pressure of carbon dioxide (PCO2) in venous blood \> 55 mmHg, or PCO2 in arterial blood \> 40 mmHg;
* Patient who has laboratory abnormalities of: creatinine \> Upper Limit of Normal (ULN), hemoglobin \< 90 g/L;
* Patient with congenital organ absence;
* Patient with a history of glucocorticoid allergy;
* Patient who is positive for human immunodeficiency (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;
* Patient who has participated in a previous gene therapy research trial;
* Patient who has any concurrent clinically significant major disease or any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.