Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

Launched by GENECRADLE INC · May 14, 2024

Keywords

ClinConnect Summary

This clinical trial is investigating a new gene therapy drug called GC101 to see if it is safe and effective for treating patients with Spinal Muscular Atrophy (SMA) Type 3. SMA is a genetic condition that affects the muscles and can lead to weakness and difficulty with movement. The study is looking for participants who are at least 2 years old and have a specific genetic diagnosis of SMA Type 3. Candidates should have a certain level of mobility, as measured by a score between 10 and 54 on the Hammersmith Functional Motor Scale, which assesses motor function in children with SMA.

If eligible, participants will receive the gene therapy through a procedure called intrathecal delivery, which means the drug is injected into the space around the spinal cord. The trial is currently recruiting individuals of any gender, but there are specific criteria that may disqualify some patients, such as previous participation in gene therapy trials, certain health conditions, or recent treatments with other SMA medications. Participants will be closely monitored for their safety and how well the treatment works. It’s important for patients and their families to understand the purpose of the study and the risks involved before deciding to join.

Gender

ALL

Eligibility criteria

• Inclusion 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.
• Exclusion Criteria:
• • 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 diseases, nervous system diseases (including but not limited to epilepsy, meningitis, history of convulsions or seizures, cerebrospinal fluid circulation disorders), and mental illnesses, etc.;
• • 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.