UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000027666
Receipt number R000031690
Scientific Title An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1
Date of disclosure of the study information 2017/07/01
Last modified on 2021/06/09 17:41:49

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Basic information

Public title

An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1

Acronym

An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1

Scientific Title

An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1

Scientific Title:Acronym

An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1

Region

Japan


Condition

Condition

progressive familial intrahepatic cholestasis type 1

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 1

Basic objectives2

Safety,Efficacy

Basic objectives -Others


Trial characteristics_1

Exploratory

Trial characteristics_2

Explanatory

Developmental phase

Not applicable


Assessment

Primary outcomes

To evaluate itching before and after 12-months treatment with sodium phenylbutylate

Key secondary outcomes

1) Physical findings
2) Biomarkers such as AST, ALT, gGTP, T.Bil, D.Bil
3) Biomarkers such as plasma amino acid fraction (isoleucine, glutamine, glutamic acid, argineine), and anmonia
4) Dermatology Life Quality Index
5) Pharmacokinetics
6) 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 12 months

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 1
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

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

6300293

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 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


Email

kondou-hiroki@med.kindai.ac.jp


Sponsor or person

Institute

Kindai University

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

Institutional Review Board of Osaka University Hospital

Address

2-15 Yamadaoka, Suita-shi, Osaka

Tel

06-6210-8290

Email

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)


Other administrative information

Date of disclosure of the study information

2017 Year 07 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

4

Results


Results date posted


Results Delayed

Delay expected

Results Delay Reason

Because it takes time to collect data.

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

2017 Year 05 Month 23 Day

Date of IRB

2017 Year 10 Month 10 Day

Anticipated trial start date

2017 Year 07 Month 01 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

2017 Year 06 Month 06 Day

Last modified on

2021 Year 06 Month 09 Day



Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000031690


Research Plan
Registered date File name
2018/11/10 PFIC1実施計画書 6.0版_180803.doc

Research case data specifications
Registered date File name

Research case data
Registered date File name