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Gene Therapy Trial Report

Summary

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


NCTID NCT06391736 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Pompe Disease (Late-onset)
Disease Ontology Term DOID:2752
Compound Name GC301
Sponsor GeneCradle Inc
Funder Type Industry
Recruitment Status
Enrollment Count 33 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant GAA
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV9
Dose 1 3.0E13 vg/kg
Dose 2 6.0E13 vg/kg
Dose 3 1.2E14 vg/kg (IIT study)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-04-25
Completion Date 2026-12
Last Update 2025-07-03

Participation Criteria


Eligible Age >=6 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
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 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);
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations Orphan Drug Designation
Recent Updates First subject dosed August 2024

Resources/Links