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ECUR-506 first-in-human study in neonatal-onset OTC deficiency
2023-506180-34-01Phase 2Authorised
- Sponsor
- iECURE
- Start
- Sep 2024
- Modality
- Gene Therapies
- Countries
- 5
01
Overview
Phase 2, open-label, single-arm trial of ECUR-506 in ornithine transcarbamylase deficiency, sponsored by iECURE. Primary endpoints: Treatment-emergent adverse events, Change from baseline in clinical and safety assessments and 1 more. Planned start Sep 2024; status: authorised.
02
Drugs
ECUR-506
03
Conditions
ornithine transcarbamylase deficiency
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