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4-phenylbutyrate    (DrugBank: 4-phenylbutyrate, Phenylbutyrate)

2 diseases
告示番号疾患名（ページ内リンク）臨床試験数
251尿素サイクル異常症1
299嚢胞性線維症2

251. 尿素サイクル異常症　［臨床試験数：42，薬物数：45（DrugBank：10），標的遺伝子数：4，標的パスウェイ数：29］
Searched query = "Urea cycle disorder", "N-acetylglutamate synthase deficiency", "NAGS deficiency", "Carbamoyl phosphate synthetase I deficiency", "CPS1 deficiency", "Ornithine transcarbamylase deficiency", "OTC deficiency", "Classic citrullinemia", "Citrullinemia type I", "Argininosuccinic aciduria", "Argininemia", "Hiperornitinemia-hiperamonemia-homocitrulinuria syndrome", "HHH syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 42 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2008-003865-23-GB
(EUCTR)		08/09/200825/07/2008A Phase 2, Switch-Over, Dose-Escalation Study of the Safety and Tolerability of HPN-100 (glyceryl tri-[4-phenylbutyrate]) Compared to Sodium Phenylbutyrate in Adult and Pediatric Subjects with Urea Cycle DisordersA Phase 2, Switch-Over, Dose-Escalation Study of the Safety and Tolerability of HPN-100 (glyceryl tri-[4-phenylbutyrate]) Compared to Sodium Phenylbutyrate in Adult and Pediatric Subjects with Urea Cycle Disorders Urea Cycle Disorder (urea cycle enzyme or transporter deficiency)
MedDRA version: 9.1;Level: LLT;Classification code 10013373;Term: Disorders of urea cycle metabolism		Product Name: glyceryl tri-(4-phenylbutyrate)(GT4P)
Product Code: HPN-100
INN or Proposed INN: NA
Other descriptive name: glyceryl tri-(4-phenylbutyrate)		Hyperion Therapeutics LimitedNULLNot RecruitingFemale: yes
Male: yes		16Phase 2United Kingdom

299. 嚢胞性線維症　［臨床試験数：1,592，薬物数：1,539（DrugBank：255），標的遺伝子数：81，標的パスウェイ数：162］
Searched query = "Cystic fibrosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 1,592 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT00590538
(ClinicalTrials.gov)		February 200327/12/2007Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygotes (for Cystic Fibrosis)A Pilot Trial of Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygous Cystic Fibrosis PatientsCystic FibrosisDrug: Sodium 4-Phenylbutyrate;Drug: Genistein (Unconjugated Isoflavones 100);Drug: PlaceboChildren's Hospital of PhiladelphiaCystic Fibrosis Foundation TherapeuticsTerminated18 YearsN/AAll9Phase 1;Phase 2United States
2NCT00016744
(ClinicalTrials.gov)		September 200131/5/2001Phenylbutyrate/Genistein Duotherapy in Delta F508-Homozygous(for Cystic Fibrosis)A Pilot Trial of Phenylbutyrate/Genistein Duotherapy in Delta F508-Homozygous Cystic Fibrosis PatientsCystic FibrosisDrug: Sodium 4-Phenylbutyrate (4PBA);Drug: Unconjugated Isoflavones 100 (PTI G-4660, 87% Genistein);Drug: PlaceboChildren's Hospital of PhiladelphiaCystic Fibrosis Foundation Therapeutics;National Center for Research Resources (NCRR)Completed18 YearsN/ABoth12Phase 1;Phase 2United States