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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-06-02

Study Completion Date

2026-12-31

Brief Summary

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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 infantile-onset Pompe disease who are younger than 6 months old will be studied.

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Detailed Description

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Conditions

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Pompe Disease Infantile-Onset

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

Single intravenous administration of GC301 at a dose of 8.0 x 10\^13 vector genomes per kilogram body weight

Group Type EXPERIMENTAL

GC301

Intervention Type GENETIC

GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene

Cohort 2

Single intravenous administration of GC301 at a dose of 1.2 x 10\^14 vector genomes per kilogram body weight

Group Type EXPERIMENTAL

GC301

Intervention Type GENETIC

GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene

Cohort 3

Single intravenous administration of GC301 at a dose of 1.8 x 10\^14 vector genomes per kilogram body weight

Group Type EXPERIMENTAL

GC301

Intervention Type GENETIC

GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene

Interventions

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GC301

GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene

Intervention Type GENETIC

Eligibility Criteria

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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.

Maximum Eligible Age

6 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No