Actively Recruiting
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
Led by GeneCradle Inc · Updated on 2025-07-03
18
Participants Needed
4
Research Sites
196 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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...
- Child is six months old or younger on the day of treatment
- Diagnosis of Spinal Muscular Atrophy Type 1 confirmed by gene mutation analysis showing bi-allelic SMN1 mutations and 2 copies of SMN2
- Onset of SMA symptoms before 6 months of age
- Legal guardian can understand the study purpose and risks and can provide signed informed consent
You will not qualify if you...
- Prior participation in any gene therapy research trial
- Previous treatment with Nusinersen or Risdiplam
- AAV9 neutralizing antibody titer equal to or greater than 1:200
- Requires non-invasive ventilatory support averaging 16 hours per day or more
- Presence of SMN2 point mutation c.859G>C
- Use of invasive ventilatory support or oxygen saturation below 95% while awake and calm at screening
- Positive for HIV antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody
- Clinically significant abnormal lab values including GGT, AST, ALT, bilirubin over 3 times normal, hemoglobin below 110 or above 150 g/L, platelet count outside 183x10^9/L to 614x10^9/L
- Classified as Class IV on Modified Ross Heart Failure Classification for Children
- History of allergy to glucocorticoids
- Contraindications to lumbar puncture
- Untreated active infection requiring systemic antiviral therapy during screening
- Vaccination within 2 weeks before vector infusion
- Any major disease or condition making participation unsuitable as judged by the investigator
- Other protocol defined inclusion/exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
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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