Evaluation of the Safety and Efficacy of Late-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

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

33 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-04-19

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 late-onset Pompe disease （LOPD） who are ≥ 6 years old will be studied.

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Detailed Description

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Conditions

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Pompe Disease (Late-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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Low dosage group

Single intravenous administration of GC301 at a dose of 3.0 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

High dosage group

Single intravenous administration of GC301 at a dose of 6.0 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

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 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 voluntarily provide signed and dated informed consent prior to any study-related procedures being performed.

Exclusion Criteria

* 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;
* Patient who has a contraindication to study drug or to corticosteroids, or has demonstrated hypersensitivity to any of the components of the study drug;
* Patient who has AAV9 neutralizing antibody titer ≥ 1:100；
* Patient who has participated in a previous gene therapy research trial;
* Pregnant or lactating female participants;
* Patients who have fertility plans within 6 months from screening to the end of the study and are unwilling to take effective physical contraceptive measures (such as a condom, intrauterine device, contraceptive ring, ligation, abstinence, etc.) for contraception (including the subject's partner);

Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No