UMIN-CTR Clinical Trial

Basic information
Public title	An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1
Acronym	An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1
Scientific Title	An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1
Scientific Title:Acronym	An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1
Region	Japan

Condition
Condition	progressive familial intrahepatic cholestasis type 1
Classification by specialty	Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Explanatory
Developmental phase	Not applicable

Assessment
Primary outcomes	To evaluate itching before and after 12-months treatment with sodium phenylbutylate
Key secondary outcomes	1) Physical findings 2) Biomarkers such as AST, ALT, gGTP, T.Bil, D.Bil 3) Biomarkers such as plasma amino acid fraction (isoleucine, glutamine, glutamic acid, argineine), and anmonia 4) Dermatology Life Quality Index 5) Pharmacokinetics 6) 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	To subscribe 450 or 600mg/kg/day sodium phenylbutylate (buphenyl) for 12 months
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)Patients who are diagnosed with PFIC type 1 2)Hospitalised- or out-patients 3)Those who can intake medicine per oral and continue the treatment 4)Those who can receive the observation necessary in this study 5) Those who have given written informed consent on the use of their data for the study (patients or legally acceptable representative)
Key exclusion criteria	1)Those who participated in another study within 3 months 2)Those who have hypersensitivity with phenylbutylate and/or its metabolite
Target sample size	6

Research contact person
Name of lead principal investigator	1st name Middle name Last name Hiroki Kondou
Organization	Kindai University Nara Hospital
Division name	Department of Pediatrics
Zip code
Address	1248-1 Otoda-cho, Ikoma, Nara, Japan
TEL	0743-77-0880
Email	kondou-hiroki@med.kindai.ac.jp

Public contact
Name of contact person	1st name Middle name Last name Hiroki Kondou
Organization	Kindai University Nara Hospital
Division name	Department of Pediatrics
Zip code
Address	1248-1 Otoda-cho, Ikoma, Nara, Japan
TEL	0743-77-0880
Homepage URL
Email	kondou-hiroki@med.kindai.ac.jp

Sponsor
Institute	Kindai University
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Government offices of other countries
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization
Address
Tel
Email

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	大阪大学医学部附属病院（大阪府）、順天堂大学附属順天堂医院（東京都）

Other administrative information
Date of disclosure of the study information	2017 Year 07 Month 01 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Open public recruiting
Date of protocol fixation	2017 Year 05 Month 23 Day
Date of IRB
Anticipated trial start date	2017 Year 07 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2017 Year 06 Month 06 Day
Last modified on	2018 Year 11 Month 10 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000031690

Research Plan
Registered date	File name
2018/11/10	PFIC1実施計画書 6.0版_180803.doc

Research case data specifications
Registered date	File name

Research case data
Registered date	File name

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