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SEL-302 in Pediatric Mut Subtype MMA
NCT05778877Phase 2Withdrawn
- Sponsor
- Selecta Biosciences
- Start
- Dec 2022
- Primary completion
- Aug 2025
- Modality
- Gene Editing, Nanoparticles, Gene Silencing
- Countries
- 1
01
Overview
Phase 2, open-label trial of SEL-302 in Methylmalonic acidemia due to methylmalonyl-CoA mutase deficiency (MMA) (2 cohorts), sponsored by Selecta Biosciences. Primary endpoints: Safety and tolerability of SEL-302 at 5 years, PD Activity of SEL-302 (sMMA levels) at 5 years and 2 more. Planned start Dec 2022; status: withdrawn.
02
Drugs
SEL-302
03
Conditions
Methylmalonic acidemia due to methylmalonyl-CoA mutase deficiency (MMA)
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