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HepaStem in pediatric urea cycle disorders
2014-000650-11Phase 2Completed
- Sponsor
- Promethera Therapeutics
- Start
- Jul 2014
- Modality
- Stem Cell Therapies → Cell Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of hepastem in urea cycle disorder (UCD), carbamoyl phosphate synthetase 1 deficiency, ornithine transcarbamylase deficiency and 5 more, sponsored by Promethera Therapeutics. Primary endpoint: Ureagenesis improvement at 6 months after first infusion. Started Jul 2014; status: completed.
02
Drugs
hepastem
03
Conditions
urea cycle disorder (UCD)carbamoyl phosphate synthetase 1 deficiencyornithine transcarbamylase deficiencycitrullinemia type 1 (CTLN1)argininosuccinic aciduria (ASA)
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