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Post-marketing Phase IV study of glycerol phenylbutyrate in Chinese pediatric urea cycle disorder patients
CTR20251147Phase 4Active
- Sponsor
- Immedica
- Start
- Dec 2025
- Modality
- Small Molecules
- Countries
- 1
01
Overview
A Phase 4 trial of glycerol phenylbutyrate in carbamoyl phosphate synthetase 1 deficiency, ornithine transcarbamylase deficiency, citrullinemia type 1 (CTLN1), argininosuccinic aciduria (ASA), arginase 1 deficiency (ARG1-D), sponsored by Immedica. Started Dec 2025; status: active.
02
Drugs
glycerol phenylbutyrate
03
Conditions
carbamoyl phosphate synthetase 1 deficiencyornithine transcarbamylase deficiencycitrullinemia type 1 (CTLN1)argininosuccinic aciduria (ASA)arginase 1 deficiency (ARG1-D)
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