Study design & results
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Trial intelligence / Profile preview
GC101 intrathecal gene therapy in infants with type 1 spinal muscular atrophy
ChiCTR2200066399Phase 2Not yet recruiting
- Sponsor
- Chinese PLA General Hospital
- Start
- Nov 2022
- Modality
- Cell Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of nolgileucel in spinal muscular atrophy type 1, sponsored by Chinese PLA General Hospital. Primary endpoints: Safety, Effectiveness and 1 more. Planned start Nov 2022; status: not yet recruiting.
02
Drugs
nolgileucel
03
Conditions
spinal muscular atrophy type 1
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