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GB221 in Pediatric Participants With Spinal Muscular Atrophy Type 1
NCT07070999Phase 2Recruiting
- Sponsor
- GEMMA Biotherapeutics
- Start
- Jan 2026
- Primary completion
- Dec 2028
- Modality
- Monoclonal Antibodies
- Countries
- 1
01
Overview
Phase 2, open-label trial of coprelotamab in spinal muscular atrophy type 1 (4 cohorts), sponsored by GEMMA Biotherapeutics. Primary endpoints: Number of participants with treatment-related adverse events (AEs) and serious adverse events (SAEs) at Grade 3 or higher at 18 months, Number of Participants with Clinically Significant Changes in Physical Functions at 18 months and 7 more. Started Jan 2026; status: recruiting.
02
Drugs
coprelotamab
03
Conditions
spinal muscular atrophy type 1
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