![]() |
UMIN-CTR Clinical Trial |
|
![]() |
![]() |
![]() |
![]() |
Name: | UMIN ID: |
Recruitment status | No longer recruiting |
Unique ID issued by UMIN | UMIN000027666 |
Receipt No. | 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 | 2020/12/08 |
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 |
|
Condition | ||
Condition | progressive familial intrahepatic cholestasis type 1 | |
Classification by specialty |
|
|
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 |
|
|
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 |
|
|||
Age-upper limit |
|
|||
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 |
|
||||||
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 | ||||||
kondou-hiroki@med.kindai.ac.jp |
Public contact | |||||||
Name of contact person |
|
||||||
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 | |
Institute | Kindai University |
Institute | |
Department |
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 |
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 |
|
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 | |
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 | No longer recruiting | ||||||
Date of protocol fixation |
|
||||||
Date of IRB |
|
||||||
Anticipated trial start date |
|
||||||
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 |
|
||||||
Last modified on |
|
Link to view the page | |
URL(English) | https://upload.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 |