臨床研究等提出・公開システム

Japanese

Date of registration	Oct. 09, 2024
Last modified on	Dec. 03, 2024
Trial ID	jRCT2041240104

Scientific Title	A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients With Primary Biliary Cholangitis (VANTAGE)
Public Title	A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis (VANTAGE)

Contact for Scientific Queries	Name	Misaki Masako
	Affiliation	Syneos Health Japan K.K.
	Address	10F Umeda Daibiru, 3-3-10 Umeda, Kita-ku, Osaka
	Telephone	+81-90-1790-0384
	E-mail	VLX-601@syneoshealth.com
Contact for Public Queries	Name	Misaki Masako
	Affiliation	Syneos Health Japan K.K.
	Address	10F Umeda Daibiru, 3-3-10 Umeda, Kita-ku, Osaka
	Telephone	+81-90-1790-0384
	E-mail	VLX-601@syneoshealth.com

Recruitment status	Recruiting

Anticipated date of first enrollment		Dec. 02, 2024
Actual date of first enrollment
Target sample size		10
Study Type		Interventional
Study Design	allocation	randomized controlled trial
	masking	double blind
	control	placebo control
	assignment	parallel assignment
	purpose	treatment purpose
Key inclusion & exclusion criteria	Inclusion Criteria	- Provide signed informed consent at the screening visit as well as comply with all study visits and requirements through the end of the study - Male or female, age >=18 years at the screening visit - Confirmed diagnosis of PBC in line with the AASLD guidelines - UDCA and anti-pruritic medication use will be allowed if meeting additional criteria - Qualified pruritus associated with PBC as assessed by Adult ItchRO
	Exclusion Criteria	- Pruritus associated with an etiology other than PBC - Evidence or clinical suspicion of decompensated cirrhosis or a history of decompensation events - Current symptomatic cholelithiasis or inflammatory gallbladder disease - History of small bowel surgery/resection impacting the terminal ileum that may disrupt the enterohepatic circulation. - Evidence, history, or suspicion of other liver diseases: PBC patients with AIH are not excluded - History of Liver transplantation
	Age Minimum	18age old over
	Age Maximum	No limit
	Gender	Both
Health Condition(s) or Problem(s) Studied		Cholestatic Pruritus in Patients with Primary Biliary Cholangitis
Intervention(s)		Volixibat (20 mg or 80 mg) or placebo is administered orally twice daily.
Health Condition(s) Keyword
Intervention(s) Keyword
Health Condition(s) Code
Intervention(s) Code
Primary Outcome(s)		Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
Secondary Outcome(s)		- Proportion of participants with itch response using the Adult ItchRO - Incidence of adverse events - Changes in alkaline phosphatase - Changes in total bilirubin - Changes in serum bile acid levels - Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40) - Change in Patient-Reported Outcomes Measurement Information System (PROMIS) fatigue questionnaire - Change in Patient-Reported Outcomes Measurement Information System (PROMIS) sleep disturbance questionnaire

Primary Sponsor	Mirum Pharmaceuticals Inc.

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support
Secondary Sponsor

Name of Certified Review Board	Hamamatsu University School of Medicine (HUSM) - Hamamatsu University Hospital Institutional Review Board
Address	1-20-1, Handayama, Chuo-ku, Hamamatsu-shi, Shizuoka, Shizuoka
Telephone
E-Mail
Approval Status	Approval
Date of approval	Oct. 08, 2024

Plan to share IPD	No
Plan description

Secondary ID(s)	NCT05050136
Issuing Authority	ClinicalTrials.gov

Countries of Recruitment（Except Japan）	US/France/Germany/Italy/Spain/China/Argentina/Brazil/Canada/Israel/UK

History of Changes

No	Publication date
2	Dec. 03, 2024	(this page)	Changes
1	Oct. 09, 2024	Detail

List of change history