Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease

Last updated: October 30, 2023
Sponsor: Seventh Medical Center of PLA General Hospital
Overall Status: Active - Recruiting

Phase

N/A

Condition

Pompe Disease

Treatment

Genetic: GC301

Clinical Study ID

NCT05567627
JLJY-GC301-IOPD-003
  • Ages < 6
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • The patient's legal guardian(s) must be able to understand the purpose and risks ofthe study and voluntarily provide signed and dated informed consent prior to anystudy-related procedures being performed;
  • The patient must be no older than 6 months;
  • The patient must be diagnosed with infantile-onset Pompe disease.

Exclusion

Exclusion Criteria:

  • Class IV patient based on Modified Ross Heart Failure Classification for Children;
  • Aspartate aminotransferase (AST), alanine aminotransferase (ALT) > 3x upper limit ofnormal (ULN), alkaline phosphatase (ALP) > 2x ULN (with the exception of liverabnormalities related to Pompe disease);
  • Patient has severe organ dysfunction, such as liver and kidney failure (Liver failure:patients may have liver failure syndrome, including fatigue, severe gastrointestinalsymptoms; clinical examination found prolonged prothrombin time, prothrombin activityless than 40%; Neuropsychiatric symptoms, such as restlessness, changes in personalityand behavior, lethargy, coma, etc.; Toxic tympanic bowel, ascites, multiple organdysfunction, etc.; hyperalbuminemia exceeding 171 μmol/L, hypoalbuminemia. Renalfailure: creatinine exceeding 110 μmol/L, or glomerular filtration rate less than 100mL/min), congenital/acquired encephalopathy, etc.;
  • Patient with congenital organ absence;
  • Patient with primary immunodeficiency;
  • Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis Bsurface antigen, hepatitis C antibody, or treponema pallidum antibody;
  • Patient with a history of glucocorticoid allergy;
  • Patient who has participated in a previous gene therapy research trial;
  • Patient who has any concurrent clinically significant major disease or any othercondition that, in the opinion of the Investigator, makes the subject unsuitable forparticipation in the study.

Study Design

Total Participants: 6
Treatment Group(s): 1
Primary Treatment: Genetic: GC301
Phase:
Study Start date:
August 01, 2022
Estimated Completion Date:
September 30, 2025

Connect with a study center

  • Bayi Children's Hospital, Seventh Medical Center, PLA general hospital

    Beijing, Beijing 100700
    China

    Active - Recruiting

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