UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Clinical trial of Bezafibrate for patients with long-chain fatty acid beta-oxydation disorder
Title of the study (Brief title)	Clinical trial of Bezafibrate for patients with long-chain fatty acid beta-oxydation disorder
Region	Japan

Condition
Condition	long-chain fatty acid beta-oxydation disorder
Classification by specialty	Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	to investigate the effects of bezafibrate on patients with long-chain fatty acid beta-oxydation disorder
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	improvement of clinical symptoms (muscle pain etc.)
Key secondary outcomes

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -but assessor(s) are blinded
Control	Historical
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	bezafibrate for internal use
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	patients with long-chain fatty acid beta-oxydation disorder
Key exclusion criteria	a case who dosen't approve this clinical trial
Target sample size	2

Research contact person
Name of lead principal investigator	Hiroyuki Awano
Organization	Kobe University Graduate School of Medicine
Division name	Psdiatrics
Address	7-5-1,Kusunokicho, Chuo, Kobe, Japan
TEL	078-382-6090
Email	awahiro@med.kobe-u.ac.jp

Public contact
Name of contact person	Hiroyuki Awano
Organization	Kobe University Graduate School of Medicine
Division name	Pediatrics
Address	7-5-1, Kusunokicho, Chuo, Kobe, Japan
TEL	078-382-6090
Homepage URL
Email	awahiro@med.kobe-u.ac.jp

Sponsor
Institute	Department of Pediatrics, Kobe University Graduate School of Medicine,
Institute
Department

Funding Source
Organization	Grant-in aid for Scientific Resaerch (KAKENHI)
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

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	2011 Year 04 Month 01 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2011 Year 02 Month 01 Day
Anticipated trial start date	2011 Year 03 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2011 Year 03 Month 16 Day
Last modified on	2018 Year 12 Month 14 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000006254