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MRT5201 in Ornithine Transcarbamylase Deficiency
NCT03767270Phase 2Withdrawn
- Sponsor
- Translate Bio
- Start
- Dec 2019
- Primary completion
- Jul 2022
- Modality
- Lipid-based Nanoparticles, mRNA Therapeutics
01
Overview
Phase 2, randomised, double-blind trial in ornithine transcarbamylase deficiency comparing MRT5201 with Placebo (2 arms). Primary endpoint: Incidence of treatment-emergent adverse events at Week 24. Planned start Dec 2019; status: withdrawn.
02
Drugs
MRT5201
03
Conditions
ornithine transcarbamylase deficiency
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