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Tests treatment safety and results for Pompe Disease (Late-onset)
Official title Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
ClinicalTrials.gov ID: NCT06391736
What this study is testing
What is GC301?
GC301 is an investigational medicine, being studied as a potential treatment for pompe disease (late-onset).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 and older
You may be able to join if
- 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...
You likely can't join if
- Patient who has any history or concurrent clinical organic disease, including cardiovascular and liver diseases, respiratory system, nervous system...
- 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...
- Patient who has AAV9 neutralizing antibody titer ≥ 1:100；
See the full eligibility 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.
- 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.