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Switch-over and long-term open-label study of HPN-100 in pediatric urea cycle disorder patients under 6 years
2014-003248-12Phase 2
- Sponsor
- Hyperion Therapeutics, Inc.
- Start
- Feb 2015
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of glycerol phenylbutyrate in urea cycle disorder (UCD), sponsored by Hyperion Therapeutics, Inc. Primary endpoint: 24-hour AUC of blood ammonia levels.
02
Drugs
glycerol phenylbutyrate
03
Conditions
urea cycle disorder (UCD)
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