DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
263	脳腱黄色腫症	1

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	NCT04270682 (ClinicalTrials.gov)	January 31, 2020	12/2/2020	Study to Evaluate Patients With Cerebrotendinous Xanthomatosis	A Phase 3 Study to Evaluate the Effects of Chenodeoxycholic Acid in Adult and Pediatric Patients With Cerebrotendinous Xanthomatosis	CTX	Drug: Blinded CDCA 250 mg TID;Drug: Placebo;Drug: Open-Label CDCA 250 mg TID;Drug: Rescue Medication CDCA 250 mg TID;Drug: CDCA Weight-Based Dose TID	Travere Therapeutics, Inc.	NULL	Recruiting	1 Month	N/A	All	12	Phase 3	United States

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

January 31, 2020

12/2/2020

Study to Evaluate Patients With Cerebrotendinous Xanthomatosis

A Phase 3 Study to Evaluate the Effects of Chenodeoxycholic Acid in Adult and Pediatric Patients With Cerebrotendinous Xanthomatosis

CTX

Drug: Blinded CDCA 250 mg TID;Drug: Placebo;Drug: Open-Label CDCA 250 mg TID;Drug: Rescue Medication CDCA 250 mg TID;Drug: CDCA Weight-Based Dose TID

Travere Therapeutics, Inc.

NULL

Recruiting

1 Month

N/A

All

Phase 3

United States