Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

Launched by GENECRADLE INC · Apr 10, 2023

Keywords

ClinConnect Summary

This clinical trial is testing a new gene therapy drug called GC101 to see if it is safe and effective for treating infants with Spinal Muscular Atrophy (SMA) Type 1. SMA is a genetic condition that affects muscle control, and this trial is specifically looking for babies who are six months old or younger and have a confirmed diagnosis of SMA with specific genetic markers. The goal is to see if delivering this therapy directly into the spinal fluid can help improve their condition.

To participate in the study, babies must meet certain criteria: they need to be diagnosed with SMA Type 1 before six months of age and have specific genetic mutations. However, there are some exclusions, such as previous participation in other gene therapy trials or certain health issues like needing prolonged breathing support. Families interested in the trial should know that their child's safety and well-being will be closely monitored throughout the study, and they will need to provide consent after understanding the risks and benefits. This trial is currently recruiting participants, and it offers a potential new treatment option for this challenging condition.

Gender

ALL

Eligibility criteria

• Inclusion 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.
• Exclusion Criteria:
• • 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), Hemoglobin (Hgb)\< 110 or \>150 g/L, platelet \<183x10\^9/L or 614x10\^9/L;
• • 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 defined inclusion/exclusion criteria may apply.