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Long-term follow-up study of intrathecal GC101 in patients with spinal muscular atrophy type 3
CTR20260891Phase 2Active
- Sponsor
- GeneCradle
- Start
- Mar 2026
- Modality
- Cell Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of nolgileucel in Spinal muscular atrophy type 3 (SMA3), sponsored by GeneCradle. Primary endpoints: Safety and immunogenicity and Long-term efficacy by scale scores. Started Mar 2026; status: active.
02
Drugs
nolgileucel
03
Conditions
Spinal muscular atrophy type 3 (SMA3)
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