DDrare: Database of Drug Development for Rare Diseases

297. アラジール症候群
［臨床試験数：26，薬物数：16（DrugBank：8），標的遺伝子数：2，標的パスウェイ数：2］

Searched query = "Alagille 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.

Drug = 3 mg [14C]-A4250 capsule; A4250; CRC (A3384); Chloride; Eicosapentaenoic acid; LUM001; MARALIXIBAT CHLORIDE; Magnesium; Magnesium gluconate; Magnesium sulfate; Maralixibat; Maralixibat (formely SHP625 or LUM001); Maralixibat chloride; Phenylbutyrate; Questran; Sulfate

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	JPRN-UMIN000012782	2014/02/01	01/02/2014	Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.		Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.	Group A; phenylbutyrate 6g (Child 100mg/kg)/day 7days Group B; phenylbutyrate 6g (Child 100mg/kg)/day 3days and 12g (Child 200mg/kg)/day 4days Group C; phenylbutyrate 6g (Child 100mg/kg)/day 1day, phenylbutyrate 12g (Child 200mg/kg)/day 2days and phenylbutyrate 21g (Child 300mg/kg)/day 4days	Juntendo University	NULL	Pending	Not applicable	Not applicable	Male and Female	2	Not selected	Japan
2	JPRN-UMIN000003802	2010/04/01	22/06/2010	Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.		Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.	phenylbutyrate(Child 250mg/kg/day)for 1-4months : phenylbutyrate(Child 350mg/kg/day)for 1-4months : phenylbutyrate(Child 500mg/kg/day)for 1-4months	Saiseikai Yokohama City Tobu Hospital	Laboratory of Molecular Pharmacokinetics, Graduate School of Pharmaceutical Sciences, The University of Tokyo (Tokyo)	Recruiting	Not applicable	Not applicable	Male and Female	15	Not selected	Japan

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

JPRN-UMIN000012782

2014/02/01

01/02/2014

Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.

Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.

Group A; phenylbutyrate 6g (Child 100mg/kg)/day *7days
Group B; phenylbutyrate 6g (Child 100mg/kg)/day *3days and 12g (Child 200mg/kg)/day *4days
Group C; phenylbutyrate 6g (Child 100mg/kg)/day *1day, phenylbutyrate 12g (Child 200mg/kg)/day *2days and phenylbutyrate 21g (Child 300mg/kg)/day *4days

Juntendo University

NULL

Pending

Not applicable

Male and Female

Not selected

Japan

JPRN-UMIN000003802

2010/04/01

22/06/2010

Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.

phenylbutyrate(Child 250mg/kg/day)for 1-4months : phenylbutyrate(Child 350mg/kg/day)for 1-4months : phenylbutyrate(Child 500mg/kg/day)for 1-4months

Saiseikai Yokohama City Tobu Hospital

Laboratory of Molecular Pharmacokinetics, Graduate School of Pharmaceutical Sciences, The University of Tokyo (Tokyo)

Recruiting

Not applicable

Male and Female

Not selected

Japan