UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000024753 |
|---|---|
| Receipt number | 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 | 2020/11/11 13:20:11 |
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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 | Hiroki |
| Middle name | |
| Last name | Kondou |
Organization
Kindai University Nara Hospital
Division name
Department of Pediatrics
Zip code
630-0293
Address
1248-1 Otoda-cho, Ikoma, Nara, Japan
TEL
0743-77-0880
kondou-hiroki@med.kindai.ac.jp
Public contact
Name of contact person
| 1st name | Hiroki |
| Middle name | |
| Last name | Kondou |
Organization
Kindai University Nara Hospital
Division name
Department of Pediatrics
Zip code
630-0293
Address
1248-1 Otoda-cho, Ikoma, Nara, Japan
TEL
0743-77-0880
Homepage URL
kondou-hiroki@med.kindai.ac.jp
Sponsor or person
Institute
Japan Agency for Medical Research and Development
Institute
Department
Personal name
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
Osaka University
Address
2-15 Yamadaoka, Suita-shi, Osaka
Tel
06-6210-8290
shiken@hp-crc.med.osaka-u.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
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
6
Results
Results date posted
Results Delayed
| Delay expected |
Results Delay Reason
Because it took a long time to meet the target number of cases.
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
No longer recruiting
Date of protocol fixation
| 2016 | Year | 09 | Month | 27 | Day |
Date of IRB
| 2016 | Year | 09 | Month | 27 | Day |
Anticipated trial start date
| 2016 | Year | 11 | Month | 18 | Day |
Last follow-up date
| 2021 | 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 | 08 | Day |
Last modified on
| 2020 | Year | 11 | Month | 11 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000028256
