UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	A study to assess the safety and efficacy of rifampicin for progressive familial intrahepatic cholestasis (PFIC) and benign recurrent intrahepatic cholestasis (BRIC)
Title of the study (Brief title)	A study to assess the safety and efficacy of rifampicin for progressive familial intrahepatic cholestasis (PFIC) and benign recurrent intrahepatic cholestasis (BRIC)
Region	Japan

Condition
Condition	Progressive familial intrahepatic cholestasis Benign recurrent intrahepatic cholestasis
Classification by specialty	Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To confirm the safety and efficacy of rifampicin therapy for progressive familial intrahepatic cholestasis and benign recurrent intrahepatic cholestasis patients, when the standard treatments of cholestasis such as ursodeoxycholic acid and biliary drainage are ineffective.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Serum bilirubin and total bile acid
Key secondary outcomes	1. Laboratory blood test data at 1, 2, 4, 8 and 12 weeks after treatment (CBC, AST, ALT, gGTP, TP, Alb, T.Chol, LDL-C, PT, APTT, Ca, P, ALP, TSH, fT4, analysis of bile acids in serum). 2. Frequency of adverse events.

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Rifampicin (RFP, 10 mg/kg/day) are given for 4-8 weeks. When RFP is effective against cholestasis, RFP will be tapered 2.5mg/kg per a week and withdrawn after 4 weeks. When bilirubin level does not decrease or the continuation of RFP is difficult for some side effects, RFP will be withdrawn.
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	Not applicable
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1. Progressive familial intrahepatic cholestasis or benign recurrent intrahepatic cholestasis patients. 2. Patients whose written informed consent has been obtained (from them or from their parents).
Key exclusion criteria	1. Patients with past histories of hypersensitivity reaction against rifampicin. 2. Patients currently receiving medications that are known to interact with rifampicin (drug for HIV infection, voriconazole, pradicantel, tadalafil, telaprevir, or simeprevir etc.) 3. Patients with serious active bacterial infection as sepsis.
Target sample size	3

Research contact person
Name of lead principal investigator	Shouichi Ohga
Organization	Graduate School of Medical Sciences, Kyushu University
Division name	Department of Pediatrics
Address	3-1-1, Maidashi, Higashi-ku, Fukuoka-city, 812-8582 Japan
TEL	092-642-5421
Email	takadah@pediatr.med.kyushu-u.ac.jp

Public contact
Name of contact person	Shunsuke Kanno
Organization	Graduate School of Medical Sciences, Kyushu University
Division name	Department of Pediatrics
Address	3-1-1, Maidashi, Higashi-ku, Fukuoka-city
TEL	092-642-5421
Homepage URL
Email	kannos@pediatr.med.kyushu-u.ac.jp

Sponsor
Institute	Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University
Institute
Department

Funding Source
Organization	Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2015 Year 06 Month 05 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2013 Year 07 Month 17 Day
Anticipated trial start date	2013 Year 07 Month 17 Day
Last follow-up date	2018 Year 12 Month 31 Day
Date of closure to data entry	2018 Year 12 Month 31 Day
Date trial data considered complete	2018 Year 12 Month 31 Day
Date analysis concluded	2018 Year 12 Month 31 Day

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results	No patient was enrolled.
Other related information

Management information
Registered date	2015 Year 06 Month 05 Day
Last modified on	2019 Year 03 Month 04 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000020647