DDrare: Database of Drug Development for Rare Diseases

263. 脳腱黄色腫症
［臨床試験数：5，薬物数：10（DrugBank：2），標的遺伝子数：2，標的パスウェイ数：4］

Searched query = "Cerebrotendinous xanthomatosis", "27-hydroxylase deficiency", "CYP27 deficiency"
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 = Blinded CDCA 250 mg TID; Blood tests; CDCA; CDCA Weight-Based Dose TID; Chenodeoxycholic Acid; Chenodeoxycholic acid; FPF1011; Lovastatin; Open-Label CDCA 250 mg TID; Rescue Medication CDCA 250 mg TID

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-JapicCTI-205157	03/8/2020	13/02/2020	An open-label study of FPF1011 in patients with cerebrotendinous xanthomatosis (phase 3)	An open-label study of FPF1011 in patients with cerebrotendinous xanthomatosis (phase 3)	Cerebrotendinous xanthomatosis	Intervention name : FPF1011 INN of the intervention : Chenodeoxycholic acid Dosage And administration of the intervention : Dose of chenodeoxycholic acid is 750 mg/day for patients aged 20 or older, and 15 mg/kg/day under ages 20, administered orally in three divided doses a day. Control intervention name : - INN of the control intervention : - Dosage And administration of the control intervention : -	Fujimoto Pharmaceutical Corporation	NULL	recruiting	0		BOTH	5	Phase 3	Japan

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Condition

Intervention

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Status

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agemin

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JPRN-JapicCTI-205157

03/8/2020

13/02/2020

An open-label study of FPF1011 in patients with cerebrotendinous xanthomatosis (phase 3)

Cerebrotendinous xanthomatosis

Intervention name : FPF1011
INN of the intervention : Chenodeoxycholic acid
Dosage And administration of the intervention : Dose of chenodeoxycholic acid is 750 mg/day for patients aged 20 or older, and 15 mg/kg/day under ages 20, administered orally in three divided doses a day.
Control intervention name : -
INN of the control intervention : -
Dosage And administration of the control intervention : -

Fujimoto Pharmaceutical Corporation

NULL

recruiting

BOTH

Phase 3

Japan