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	NCT02963077 (ClinicalTrials.gov)	July 2013	1/11/2016	A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384	A Phase I, Double-Blind Single and Multiple Ascending Dose Study to Assess Safety and Pharmacokinetics of A4250 as Monotherapy, and in Combination With Colonic Release Cholestyramine (A3384) or Commercially Available Cholestyramine (Questran™) in Healthy Subjects	Orphan Cholestatic Liver Diseases;Primary Biliary Cirrhosis;Progressive Familial Intrahepatic Cholestasis;Alagille Syndrome	Drug: A4250;Drug: CRC (A3384);Drug: Questran;Drug: Placebo	Albireo	NULL	Completed	18 Years	60 Years	Both	94	Phase 1	NULL

No.

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Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

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Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
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NCT02963077
(ClinicalTrials.gov)

July 2013

1/11/2016

A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384

A Phase I, Double-Blind Single and Multiple Ascending Dose Study to Assess Safety and Pharmacokinetics of A4250 as Monotherapy, and in Combination With Colonic Release Cholestyramine (A3384) or Commercially Available Cholestyramine (Questran™) in Healthy Subjects

Orphan Cholestatic Liver Diseases;Primary Biliary Cirrhosis;Progressive Familial Intrahepatic Cholestasis;Alagille Syndrome

Drug: A4250;Drug: CRC (A3384);Drug: Questran;Drug: Placebo

Albireo

NULL

Completed

18 Years

60 Years

Both

Phase 1

NULL