UMIN-CTR Clinical Trial

Basic information
Public title	A pharmacokinetic study of sodium phenylbutyrate in refractory pediatric cholestasis disease
Acronym	A pharmacokinetic study of sodium phenylbutyrate in refractory pediatric cholestasis disease
Scientific Title	A pharmacokinetic study of sodium phenylbutyrate in refractory pediatric cholestasis disease
Scientific Title:Acronym	A pharmacokinetic study of sodium phenylbutyrate in refractory pediatric cholestasis disease
Region	Japan

Condition
Condition	Refractory pediatric cholestasis disease (progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis, Alagille syndrome, drug-induced cholestatic liver injury, biliary atresia, etc)
Classification by specialty	Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To clarify pharmacokinetic properties of sodium phenylbutyrate and its metabolites in refractory pediatric cholestasis disease
Basic objectives2	PK,PD
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Explanatory
Developmental phase	Phase I

Assessment
Primary outcomes	To determine concentration of phenylbutylate and its metabolites in blood and urine after oral administration of sodium phenylbutyrate
Key secondary outcomes	1) Plasma amino acid fraction (isoleucine, glutamine, glutamic acid, argineine), and anmonia 2) 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 administer sodium phenylbutylate orally at less than 200mg/kg.
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	6 months-old <=
Age-upper limit	216 months-old >
Gender	Male and Female
Key inclusion criteria	1) Subjects with diagnosis of refractory pediatric cholestasis disease. 2) Hospitalised patients. 3) Those who can intake medicine orally. 4) Those who can receive the observation essential for this study. 5) Subjects who have been determined eligible by principal investigator or sub-investigator from physical and clinical examinations. 6) Those who have agreed with participating in this study by written informed consent prior to the study (patients or legally acceptable representative).
Key exclusion criteria	1) Those who have hypersensitivity with phenylbutylate, its metabolites, drug additives and/or benzoate, etc 2) Subjects with congestive heart failure, kidney failure, and/or hypernatremia with edema 3) Subjects with renal dysfunction 4) Subjects with liver cirrhosis 5) Subjects who have been determined ineligible by principal investigator or sub-investigator
Target sample size	10

Research contact person
Name of lead principal investigator	1st name Kei Middle name Last name Minowa
Organization	Juntendo University, Faculty of Medicine
Division name	Department of Pediatrics
Zip code	113-8421
Address	2-1-1, Hongo, Bunkyo-ward, Tokyo
TEL	03-3813-3111
Email	kminowa@juntendo.ac.jp

Public contact
Name of contact person	1st name Kei Middle name Last name Minowa
Organization	Juntendo University, Faculty of Medicine
Division name	Department of Pediatrics
Zip code	113-8421
Address	2-1-1, Hongo, Bunkyo-ward, Tokyo
TEL	03-3813-3111
Homepage URL
Email	kminowa@juntendo.ac.jp

Sponsor
Institute	Juntendo 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	Japan

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

IRB Contact (For public release)
Organization	GCP center, Juntendo University Hospital
Address	3-1-3, Hongo, Bunkyo-ward, Tokyo
Tel	03-3813-3111
Email	kenkyu5858@juntendo.ac.jp

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	順天堂大学医学部附属順天堂医院（東京都）、神栖済生会病院（茨城県）、東京大学　大学院薬学系研究科　分子薬物動態学教室（東京都）、千葉こども病院（千葉県）、藤田保健衛生大学病院（愛知県）、大阪大学医学部附属病院（大阪府）、済生会横浜市東部病院（神奈川県）

Other administrative information
Date of disclosure of the study information	2016 Year 11 Month 29 Day

Related information
URL releasing protocol
Publication of results	Published

Result
URL related to results and publications
Number of participants that the trial has enrolled	7
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	Completed
Date of protocol fixation	2016 Year 11 Month 14 Day
Date of IRB	2017 Year 10 Month 19 Day
Anticipated trial start date	2016 Year 11 Month 14 Day
Last follow-up date	2020 Year 03 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2016 Year 11 Month 29 Day
Last modified on	2021 Year 12 Month 03 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000028807