Unique ID issued by UMIN | UMIN000027666 |
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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 |
An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1
An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1
An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1
An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1
Japan |
progressive familial intrahepatic cholestasis type 1
Pediatrics |
Others
NO
To evaluate the efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1
Safety,Efficacy
Exploratory
Explanatory
Not applicable
To evaluate itching before and after 12-months treatment with sodium phenylbutylate
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
Interventional
Single arm
Non-randomized
Open -no one is blinded
Uncontrolled
1
Treatment
Medicine |
To subscribe 450 or 600mg/kg/day sodium phenylbutylate (buphenyl) for 12 months
Not applicable |
Not applicable |
Male and Female
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)
1)Those who participated in another study within 3 months
2)Those who have hypersensitivity with phenylbutylate and/or its metabolite
6
1st name | Hiroki |
Middle name | |
Last name | Kondou |
Kindai University Nara Hospital
Department of Pediatrics
6300293
1248-1 Otoda-cho, Ikoma, Nara, Japan
0743-77-0880
kondou-hiroki@med.kindai.ac.jp
1st name | Hiroki |
Middle name | |
Last name | Kondou |
Kindai University Nara Hospital
Department of Pediatrics
630-0293
1248-1 Otoda-cho, Ikoma, Nara, Japan
0743-77-0880
kondou-hiroki@med.kindai.ac.jp
Kindai University
Japan Agency for Medical Research and Development
Government offices of other countries
Institutional Review Board of Osaka University Hospital
2-15 Yamadaoka, Suita-shi, Osaka
06-6210-8290
shiken@hp-crc.med.osaka-u.ac.jp
NO
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)
2017 | Year | 07 | Month | 01 | Day |
Unpublished
4
Delay expected |
Because it takes time to collect data.
No longer recruiting
2017 | Year | 05 | Month | 23 | Day |
2017 | Year | 10 | Month | 10 | Day |
2017 | Year | 07 | Month | 01 | Day |
2020 | Year | 03 | Month | 31 | Day |
2017 | Year | 06 | Month | 06 | Day |
2021 | Year | 06 | Month | 09 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000031690
Research Plan | |
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Registered date | File name |
2018/11/10 | PFIC1実施計画書 6.0版_180803.doc |
Research case data specifications | |
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Registered date | File name |
Research case data | |
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Registered date | File name |