Study Summary
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.
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GC101 adeno-associated virus injectionGENETIC
Self-complementary recombinant adeno-associated viral vector (scAAV) containing a single-stranded transgene encoding a codon-optimized human SMN1 gene
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| The Seventh Medical Center of Chinese PLA General Hospital | Beijing | China | |
| Children's Medical Center of Peking University First Hospital | Beijing | China | |
| Beijing Children's Hospital, Capital Medical University | Beijing | China | |
| National Children's Medical Center，Shanghai Jiaotong University | Shanghai | China | |
| Shenzhen Children's Hospital | Shenzhen | China | |
| Children's Hospital of Soochow University | Suzhou | China | |
| Wuhan Children's Hospital, Tongji Medical College, Huazhong University of Science & Technology | Wuhan | China |