Condition: SMA - Spinal Muscular Atrophy · Sponsor: GeneCradle Inc
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.
This description comes directly from the study's public registry record.
GeneCradle, Inc China · +8613501380583 · ind@bj-genecradle.com
Always discuss trial participation with your own doctor first.
| The Seventh Medical Center of Chinese PLA General Hospital | Beijing, China | Recruiting |
| Children's Medical Center of Peking University First Hospital | Beijing, China | Recruiting |
| Beijing Children's Hospital, Capital Medical University | Beijing, China | Recruiting |
| National Children's Medical Center，Shanghai Jiaotong University | Shanghai, China | Recruiting |
| Shenzhen Children's Hospital | Shenzhen, China | Recruiting |
| Children's Hospital of Soochow University | Suzhou, China | Recruiting |
| Wuhan Children's Hospital, Tongji Medical College, Huazhong University of Science & Technology | Wuhan, China | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06971094