Actively Recruiting
Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in Treating Spinal Muscular Atrophy Type 1 Patients in a Multi-center, Open-label, Dose Escalation Trial
Led by GeneCradle Inc · Updated on 2025-07-03
18
Participants Needed
4
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of the gene therapy drug GC101 for treating patients with Spinal Muscular Atrophy Type 1 (SMA 1). This open-label, dose-escalation clinical trial is conducted at multiple centers in China and focuses on children diagnosed with SMA 1, a condition caused by specific genetic mutations. The study aims to understand how well the treatment works in helping patients reach motor milestones and to monitor the safety of the therapy over time. Participants receive a one-time dose of GC101 delivered through an intrathecal injection, which means the drug is administered directly into the spinal fluid. There are two dosage groups: a low dose group receiving 2.4x10^14 viral genomes per person and a high dose group receiving 4.8x10^14 viral genomes per person, each with three patients. The study includes a 52-week short-term safety evaluation followed by an optional long-term follow-up lasting five years. During the trial, patients will have assessments at the start and at various points afterward to monitor their progress. Researchers will track the occurrence of any treatment-related side effects and evaluate if patients can sit without support for at least 10 seconds by 18 months of age. Other measures include survival without major events, ability to thrive, and changes in neuromuscular function scores. The total participation time may extend up to five years for those continuing in the long-term follow-up phase.
CONDITIONS
Brief Title
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Six months of age and younger on day of vector infusion with Type 1 SMA
- Diagnosis of SMA confirmed by gene mutation analysis showing bi-allelic SMN1 mutations and 2 copies of SMN2
- Onset of disease before 6 months of age
- Legal guardian able to understand study purpose and risks and provide signed informed consent
You will not qualify if you...
- Previous participation in gene therapy research trials
- Prior treatment with Nusinersen or Risdiplam
- AAV9 neutralizing antibody titer ≥1:200
- Requires non-invasive ventilatory support averaging 16 hours or more per day
- Presence of SMN2 point mutation (c.859G>C)
- Use of invasive ventilatory support or low oxygen saturation (<95%) while awake and calm
- Positive for HIV, hepatitis B, hepatitis C, or syphilis antibodies
- Abnormal lab values including elevated liver enzymes or abnormal hemoglobin and platelet counts
- Class IV heart failure according to Modified Ross Heart Failure Classification
- History of glucocorticoid allergy
- Contraindications to lumbar puncture
- Active untreated infection requiring antiviral therapy
- Vaccination less than 2 weeks before vector infusion
- Other major diseases or conditions making participation unsuitable as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - One-time administration with 52 weeks of short-term safety evaluation
Participants receive a one-time intrathecal administration of the gene therapy drug GC101 at one of two dosage levels.
Baseline visit and multiple follow-up visits during 52 weeks
Duration - Up to 5 years
Participants enter a long-term follow-up study to monitor safety and efficacy for up to 5 years after treatment.
Periodic follow-up visits as scheduled
Trial Site Locations
Total: 4 locations
1
Peking University， First Hospital, Department of Pediatrics
Beijing, China, 100034
Actively Recruiting
2
Bayi Children's Hospital, Seventh Medical Center, PLA general hospital
Beijing, China, 100700
Actively Recruiting
3
Shengjing Hospital of China Medical University
Shenyang, China, 110004
Actively Recruiting
4
Children's Hospital of Soochow University
Suzhou, China, 215025
Actively Recruiting
Research Team
G
GeneCradle, Inc. China
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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