Gene therapy for late-onset Pompe disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study evaluates the safety and effectiveness of a gene therapy drug called GC301, which uses an adeno-associated virus vector to deliver a codon-optimized version of the human acid alpha-glucosidase gene. It targets patients diagnosed with late-onset Pompe disease who are at least 6 years old. The study will assess the drug's impact on patients' respiratory function and overall health. Participants must meet specific criteria, including certain levels of lung function and mobility.

Why it matters

Who can join: the full rules

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);

Where it's running

• Chinese PLA General Hospital
  Beijing, China Recruiting

Study contacts

• Principal investigator: Guang Yang — Chinese PLA General Hospital
• Study coordinator: GeneCradle, Inc China
• Email: ind@bj-genecradle.com
• Phone: 86-13501380583

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• AB-1009 gene therapy for adults with late-onset Pompe disease

  This study tests a one-time IV gene therapy (AB-1009) to try to restore GAA enzyme activity and improve muscle and breathing function in adults with late-onset Pompe disease who are already on enzyme replacement therapy.

  Recruiting at 5 sites Phase 1/2 AskBio Inc · Phoenix, Arizona and 8 other locations · NCT07282847

• Tracking pregnancy outcomes in women with Pompe disease

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  Recruiting at 34 sites Sanofi · Phoenix, Arizona and 34 other locations · NCT00567073

• Gene therapy for treating Type 2 Spinal Muscular Atrophy

  This study is testing a new gene therapy called GC101 to see if it can help children aged 2 to 12 with Type 2 Spinal Muscular Atrophy feel better compared to their current treatment.

  Recruiting at 7 sites Phase 3 GeneCradle Inc · Beijing and 6 other locations · NCT06971094

• Gene therapy for treating spinal muscular atrophy type 3

  This study is testing a new gene therapy for people with spinal muscular atrophy type 3 to see if it is safe and helps improve their movement over time.

  Recruiting Phase 1/2 GeneCradle Inc · Beijing, Beijing Municipality · NCT06421831

• Evaluating a gene therapy drug for patients with high triglycerides and pancreatitis

  This study is testing a new gene therapy drug to see if it can help people with high triglycerides and a history of pancreatitis feel better when other treatments haven't worked.

  Recruiting Phase 1 GeneCradle Inc · Beijing, Beijing Municipality · NCT05860569

• Gene therapy for treating spinal muscular atrophy Type 1

  This study is testing a new gene therapy for babies with spinal muscular atrophy Type 1 to see if it helps them sit up on their own by 18 months old.

  Recruiting at 4 sites Phase 1/2 GeneCradle Inc · Beijing and 3 other locations · NCT05824169

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.