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UMIN ID:

Recruitment status Open public recruiting
Unique ID issued by UMIN UMIN000024753
Receipt No. R000028256
Scientific Title A clinical trial of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 2
Date of disclosure of the study information 2016/12/01
Last modified on 2017/11/10

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Basic information
Public title A clinical trial of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 2
Acronym A clinical trial of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 2
Scientific Title A clinical trial of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 2
Scientific Title:Acronym A clinical trial of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 2
Region
Japan

Condition
Condition progressive familial intrahepatic cholestasis type 2
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 2
Basic objectives2 Safety,Efficacy
Basic objectives -Others
Trial characteristics_1 Exploratory
Trial characteristics_2 Explanatory
Developmental phase Phase II

Assessment
Primary outcomes To evaluate liver histopathology before and after 24-weeks treatment with sodium phenylbutylate
Key secondary outcomes 1)Liver histopathological score
2)Reliability of histopathological scoring system
3)Biomarkers such as AST, ALT, g-GTP, T.Bil, D.Bil
4)Itching
5)Biomarkers such as plasma amino acid fraction (isoleucine, glutamine, glutamic acid, argineine), and anmonia
6)Survival rate
7)Liver transplantation avoidance rate
8)Pharmacokinetics
9)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 24 weeks
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 2
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
3)Those who received liver transplantation
4)Those who received biliary diversion
5)Those who are pregnant or hoping pregnancy
6)Those who cannot accept to practice contraception
7)Those with congestive heart failure
8)Those with renal dysfunction
9)Those with nonsense or frameshift mutation, or exon deletion in both ABCB11 alleles
10) Those with liver cirrhosis
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 Japan Agency for Medical Research and Development
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)、久留米大学病院(福岡県)、鳥取大学医学部附属病院(鳥取県)、宮城県立こども病院(宮城県)

Other administrative information
Date of disclosure of the study information
2016 Year 12 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
2016 Year 09 Month 27 Day
Date of IRB
Anticipated trial start date
2016 Year 11 Month 18 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
2016 Year 11 Month 08 Day
Last modified on
2017 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=R000028256

Research Plan
Registered date File name
2018/11/10 FIX_PFIC2 _実施計画書_ 第10.0.0版_181004.pdf

Research case data specifications
Registered date File name

Research case data
Registered date File name


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