NCT05824169 Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
| NCT ID | NCT05824169 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | GeneCradle Inc |
| Condition | Spinal Muscular Atrophy |
| Study Type | INTERVENTIONAL |
| Enrollment | 18 participants |
| Start Date | 2023-02-25 |
| Primary Completion | 2026-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 18 participants in total. It began in 2023-02-25 with a primary completion date of 2026-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05824169 clinical trial?
This trial is open to participants of all sexes, aged 0 Months or older, up to 6 Months, studying Spinal Muscular Atrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05824169 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05824169 currently recruiting?
Yes, NCT05824169 is actively recruiting participants. Contact the research team at ind@bj-genecradle.com for enrollment information.
Where is the NCT05824169 trial being conducted?
This trial is being conducted at Beijing, China, Beijing, China, Shenyang, China, Suzhou, China.
Who is sponsoring the NCT05824169 clinical trial?
NCT05824169 is sponsored by GeneCradle Inc. The trial plans to enroll 18 participants.