UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000012782 |
|---|---|
| Receipt number | R000014934 |
| Scientific Title | Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. |
| Date of disclosure of the study information | 2014/02/01 |
| Last modified on | 2014/01/12 13:33:30 |
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Basic information
Public title
Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.
Acronym
Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.
Scientific Title
Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.
Scientific Title:Acronym
Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.
Region
| Japan |
Condition
Condition
Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.
Classification by specialty
| Hepato-biliary-pancreatic medicine | Pediatrics |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
subjective and objective symptom (pruritus, jaundice)
T-Bil, D-Bil, AST, ALT, LDH, ALP, TBA, urine pH, glucose, occult blood, bilirubin, urobilinogen.
Key secondary outcomes
Base
Study type
Interventional
Study design
Basic design
Parallel
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
3
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Group A; phenylbutyrate 6g (Child 100mg/kg)/day *7days
Interventions/Control_2
Group B; phenylbutyrate 6g (Child 100mg/kg)/day *3days and 12g (Child 200mg/kg)/day *4days
Interventions/Control_3
Group C; phenylbutyrate 6g (Child 100mg/kg)/day *1day, phenylbutyrate 12g (Child 200mg/kg)/day *2days and phenylbutyrate 21g (Child 300mg/kg)/day *4days
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
Patients with caretaker's approval
Key exclusion criteria
History of drug hypersensitivity
Severe liver injury
Renal function impairment
Severe complication
Poor medical compliance
Target sample size
2
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Mitsuyoshi Suzuki |
Organization
Juntendo University
Division name
Department of Pediatrics and Adolescent Medicine
Zip code
Address
Hongo 2-1-1, Bunkyo-ku, Tokyo 113-8421
TEL
+81-(0)3-3813-3111(3325)
msuzuki@juntendo.ac.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Nakayuki Naritaka |
Organization
Juntendo University
Division name
Department of Pediatrics and Adolescent Medicine
Zip code
Address
Hongo 2-1-1, Bunkyo-ku, Tokyo 113-8421
TEL
+81-(0)3-3813-3111(3325)
Homepage URL
naritaka@juntendo.ac.jp
Sponsor or person
Institute
Juntendo University
Institute
Department
Personal name
Funding Source
Organization
MEXT scientific research fund
Organization
Division
Category of Funding Organization
Nationality of Funding Organization
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Address
Tel
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
| 2014 | Year | 02 | 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
Preinitiation
Date of protocol fixation
| 2012 | Year | 04 | Month | 01 | Day |
Date of IRB
Anticipated trial start date
| 2014 | Year | 02 | 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
| 2014 | Year | 01 | Month | 07 | Day |
Last modified on
| 2014 | Year | 01 | Month | 12 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000014934
