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Tests treatment safety and results for Spinal Muscular Atrophy Type 3
Official title Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
ClinicalTrials.gov ID: NCT06421831
What this study is testing
What is GC101?
GC101 is an investigational medicine, being studied as a potential treatment for spinal muscular atrophy type 3.
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
- The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.
- 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 2 and older
You may be able to join if
- ≥2 years of age on the day of signing the informed consent form;
- Genetic and clinical diagnosis of type 3 SMA with bi-allelic deletion of SMN1 of 5qSMA;
- Hammersmith Functional Motor Scale - Expanded (HFMSE) score is between 10 and 54 at screening;
- Female patients of childbearing age who are pregnant or lactating, as well as all enrolled patients （both male and female), should take effective...
- Patients or patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated...
You likely can't join if
- Patient who has participated in any previous gene therapy research trials;
- Patient who has AAV9 neutralizing antibody titer ≥1:200;
- Patient who has received Nusinersen within 120 days and Risdiplam within 15 days before treatment;
- Patient who requires invasive or non-invasive ventilatory support averaging≥16 hours/day at screening;
- SMN2 copy numbers \>4；
- Patient who needs nasal or gastric tube feeding for eating;
See the full eligibility criteria
- ≥2 years of age on the day of signing the informed consent form;
- Genetic and clinical diagnosis of type 3 SMA with bi-allelic deletion of SMN1 of 5qSMA;
- Hammersmith Functional Motor Scale - Expanded (HFMSE) score is between 10 and 54 at screening;
- Female patients of childbearing age who are pregnant or lactating, as well as all enrolled patients （both male and female), should take effective contraceptive measures within 6 months after the treatment;
- Patients or 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 participated in any previous gene therapy research trials;
- Patient who has AAV9 neutralizing antibody titer ≥1:200;
- Patient who has received Nusinersen within 120 days and Risdiplam within 15 days before treatment;
- Patient who requires invasive or non-invasive ventilatory support averaging≥16 hours/day at screening;
- SMN2 copy numbers \>4；
- Patient who needs nasal or gastric tube feeding for eating;
- Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;
- Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients
- Severe contractures at screening that interfere with either the ability to attain/demonstrate functional measures or with the ability to receive intrathecal (IT) dosing;
- Patient who has other serious diseases, such as severe cardiovascular and cerebrovascular diseases, digestive system diseases, urinary system diseases, endocrine system diseases, hematological diseases, immune system...
- Patient with previous injuries (such as upper or lower limb fractures) or surgical operations that have not fully recovered or reached a stable state;
- Vaccination no longer than 2 weeks before treatment;
- Patient who has any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, Beijing Municipality, 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, 2 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, Beijing Municipality, 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.