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Tests treatment safety and results for Spinal Muscular Atrophy
Official title Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
ClinicalTrials.gov ID: NCT05824169
What this study is testing
What is GC101?
GC101 is an investigational medicine, being studied as a potential treatment for spinal muscular atrophy.
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 1 (SMA 1) 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 0 to 6
You may be able to join if
- Six months of age and younger on day of vector infusion with Type 1 SAM as defined by the following features:
- Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 2 copies of SMN2;
- Onset of disease before 6 months of age
- 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 participated in a previous gene therapy research trials;
- Patient who has received Nusinersen and Risdiplam treatment;
- Patient who has AAV9 neutralizing antibody titer ≥1:200;
- Patient who requires non-invasive ventilatory support averaging≥16 hours/day;
- Patient with a point mutation in SMN2 (c.859G\>C);
- Patient who requires non-invasive ventilatory support averaging≥16 hours/day at screening;
See the full eligibility criteria
- Six months of age and younger on day of vector infusion with Type 1 SAM as defined by the following features:
- Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 2 copies of SMN2;
- Onset of disease before 6 months of age
- 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 participated in a previous gene therapy research trials;
- Patient who has received Nusinersen and Risdiplam treatment;
- Patient who has AAV9 neutralizing antibody titer ≥1:200;
- Patient who requires non-invasive ventilatory support averaging≥16 hours/day;
- Patient with a point mutation in SMN2 (c.859G\>C);
- Patient who requires non-invasive ventilatory support averaging≥16 hours/day at screening;
- Patient who use invasive ventilatory support or pulse oximetry \< 95% saturation while awake and calm at screening;
- Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;
- Abnormal laboratory values considered clinically significant, including gamma-glutamyl transferase(GGT), Aspartate aminotransferase (AST), alanine aminotransferase (ALT), bilirubin \> 3x upper limit of normal (ULN)...
- Class IV patient based on Modified Ross Heart Failure Classification for Children;
- Patient with a history of glucocorticoid allergy;
- Contraindication that would interfere with the lumbar puncture procedures;
- Presence of an untreated active infection requiring systemic antiviral therapy at any time during the screening period;
- Vaccination less than 2 weeks before infusion of vector;
- Patient who has any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study. Note: Other protocol...
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, China
- Shenyang, China
- Suzhou, 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, 0 months to 6 months. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, China; Shenyang, China; Suzhou, 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.