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Open, Prospective, Historic-Controlled, Multicenter Study of Heparesc (HHLivC) in Neonatal-Onset Urea Cycle Disorders
2015-000988-14Phase 2
- Sponsor
- Promethera Therapeutics
- Start
- Oct 2016
- Modality
- Cell Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of Heparesc in ornithine transcarbamylase deficiency, carbamoyl phosphate synthetase 1 deficiency and citrullinemia type 1 (CTLN1), sponsored by Promethera Therapeutics. Primary endpoint: Change in 13C urea formation.
02
Drugs
Heparesc
03
Conditions
ornithine transcarbamylase deficiencycarbamoyl phosphate synthetase 1 deficiencycitrullinemia type 1 (CTLN1)
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