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)

Email

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


Email

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


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



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


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name