Actively Recruiting
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
Led by GeneCradle Inc · Updated on 2025-07-03
21
Participants Needed
1
Research Sites
238 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 3 (SMA 3) patients.
CONDITIONS
Official Title
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- At least 2 years old on the day of signing informed consent
- Genetic and clinical diagnosis of type 3 SMA with bi-allelic deletion of SMN1 of 5qSMA
- Hammersmith Functional Motor Scale - Expanded (HFMSE) score between 10 and 54 at screening
- Female patients of childbearing age who are pregnant or lactating, and all patients, must use effective contraception for 6 months after treatment
- Patients or legal guardians must understand study purpose and risks and provide signed informed consent
You will not qualify if you...
- Previous participation in any gene therapy research trials
- AAV9 neutralizing antibody titer of 1:200 or higher
- Received Nusinersen within 120 days or Risdiplam within 15 days before treatment
- Requires invasive or non-invasive ventilatory support averaging 16 hours/day or more at screening
- SMN2 copy number greater than 4
- Needs nasal or gastric tube feeding for eating
- Positive for HIV antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody
- Known allergy or hypersensitivity to prednisolone, other glucocorticosteroids, or their components
- Severe contractures interfering with functional measures or intrathecal dosing
- Other serious diseases including severe cardiovascular, cerebrovascular, digestive, urinary, endocrine, hematological, immune, nervous system disorders, or mental illnesses
- Unhealed or unstable previous injuries or surgical operations
- Vaccination within 2 weeks before treatment
- Any other condition making the patient unsuitable in investigator's opinion
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Beijing Tiantan Hospital, Capital Medical University
Beijing, Beijing Municipality, China
Actively Recruiting
Research Team
G
GeneCradle, Inc China
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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