Actively Recruiting
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
Led by GeneCradle Inc · Updated on 2025-06-05
50
Participants Needed
7
Research Sites
83 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen. The primary objective of the trial is to evaluate the efficacy of GC101 in treating Type 2 SMA. The secondary objectives are to assess the efficacy, safety, and pharmacokinetic (PK) profile of GC101 in treating Type 2 SMA.
CONDITIONS
Official Title
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of Type 2 5q-Spinal Muscular Atrophy by clinical and genetic testing
- Regular nusinersen treatment for more than one year before screening
- No risdiplam treatment within 2 months before screening and no plans for risdiplam treatment within 12 months after enrollment
- Ability to sit independently but unable to walk independently at screening, with HFMSE score of 10 points or more
- Patients and/or guardians able to understand and willing to comply with trial procedures and provide informed consent
You will not qualify if you...
- Serum anti-AAV9 neutralizing antibody titers greater than 1:50 at screening
- Nusinersen treatment within 2 months prior to enrollment
- Medical conditions affecting study results or participant safety, including organ dysfunction, acute infections, immunodeficiency, severe cardiovascular/cerebrovascular diseases, gastrointestinal diseases, diabetes, epilepsy, meningitis, seizures, psychiatric disorder family history, or cerebrospinal fluid circulation disorders
- Severe liver injury or hepatic insufficiency (ALT or AST ≥3 times ULN; total bilirubin ≥1.5 times ULN)
- Contraindications to glucocorticoid use such as severe hypertension, diabetes, infections, glaucoma, osteoporosis, peptic ulcers, tuberculosis
- Contraindications to lumbar puncture or intrathecal injection
- Conditions affecting motor function assessment such as severe scoliosis, joint contractures, planned spinal surgery during trial, severe osteoporosis, or fracture history
- Positive for hepatitis B surface antigen, HIV antibodies, hepatitis C virus antibodies, or syphilis antibodies
- Vaccination within 2 weeks prior to dosing
- Previous gene therapy or participation in any clinical trial within 3 months prior to screening
- Investigator judgment deeming the patient unsuitable for study participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 7 locations
1
The Seventh Medical Center of Chinese PLA General Hospital
Beijing, China, 100700
Actively Recruiting
2
Children's Medical Center of Peking University First Hospital
Beijing, China, 102699
Actively Recruiting
3
Beijing Children's Hospital, Capital Medical University
Beijing, China
Actively Recruiting
4
National Children's Medical Center，Shanghai Jiaotong University
Shanghai, China, 200127
Actively Recruiting
5
Shenzhen Children's Hospital
Shenzhen, China
Actively Recruiting
6
Children's Hospital of Soochow University
Suzhou, China
Actively Recruiting
7
Wuhan Children's Hospital, Tongji Medical College, Huazhong University of Science & Technology
Wuhan, China, 430015
Actively Recruiting
Research Team
G
GeneCradle, Inc China
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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