Actively Recruiting
A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy Type 3 Patients
Led by GeneCradle Inc · Updated on 2025-07-03
21
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of a gene therapy drug called GC101 for patients with Spinal Muscular Atrophy Type 3 (SMA 3). This open-label, dose-escalation trial is being conducted across multiple centers in China to understand how this treatment impacts muscle function in this patient group. GC101 is delivered as a one-time dose directly into the spinal fluid through an intrathecal injection at a dose of 1.2x10^14 viral genomes per person. The study has a short-term safety evaluation over 52 weeks, after which patients may enter a long-term follow-up period lasting up to 5 years. The primary measure of effectiveness is the change from baseline in motor function scores, specifically the Hammersmith Functional Motor Scale Expanded (HFMSE) and Revised Upper Limb Module (RULM) for patients aged 6 years and older, assessed 12 months after treatment. Participants will be tested at the start of the study and followed up at various scheduled times to monitor safety and motor function. Researchers will track treatment-emergent adverse events during the 52 weeks and measure changes in motor abilities using standardized scales. The study focuses on careful monitoring of safety and motor improvements, with participation lasting up to 5 years for those who continue in long-term follow-up.
CONDITIONS
Brief 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...
- 2 years of age or older on the day of signing the informed consent form
- Genetic and clinical diagnosis of type 3 Spinal Muscular Atrophy with bi-allelic deletion of SMN1 gene
- 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
- Ability to understand the study purpose and risks and provide signed informed consent
You will not qualify if you...
- Participation in any previous 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 or more per day
- SMN2 copy numbers 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 or other glucocorticosteroids or their ingredients
- Severe contractures interfering with functional measures or intrathecal dosing
- Presence of serious diseases like severe cardiovascular, cerebrovascular, digestive, urinary, endocrine, hematological, immune system diseases, nervous system diseases including epilepsy or seizures, or mental illnesses
- Previous injuries or surgeries not fully recovered or stable
- Vaccination within 2 weeks before treatment
- Any other condition 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 - 1 day
Participants receive a one-time intrathecal administration of the gene therapy drug GC101.
1 treatment visit (in-person)
Duration - 52 weeks
Participants are monitored for safety and efficacy outcomes for 52 weeks after treatment.
Multiple follow-up visits throughout 52 weeks
Duration - Up to 5 years
Participants may enter a long-term follow-up study to monitor safety and efficacy for up to 5 years after the initial 52-week follow-up.
Follow-up visits at scheduled intervals during long-term monitoring
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
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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