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Name:
UMIN ID:

Recruitment status Completed
Unique ID issued by UMIN UMIN000016951
Receipt No. R000019676
Scientific Title The multicenter continuous clinical trial of the safety and efficacy of bezafibrate for mitochondrial fatty acid beta-oxidation disorders
Date of disclosure of the study information 2015/04/01
Last modified on 2015/11/24

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Basic information
Public title The multicenter continuous clinical trial of the safety and efficacy of bezafibrate for mitochondrial fatty acid beta-oxidation disorders
Acronym BezFAOD continuous study
Scientific Title The multicenter continuous clinical trial of the safety and efficacy of bezafibrate for mitochondrial fatty acid beta-oxidation disorders
Scientific Title:Acronym BezFAOD continuous study
Region
Japan

Condition
Condition mitochondrial fatty acid beta-oxidation disorders
Classification by specialty
Pediatrics
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 To Verify the existence and its contents of adverse events and side effects caused by continuous administration of bezafibrate by the multicenter uncontrolled open-label trial for the subjects who completed the preceding study (HUPE-002-01) for the mitochondrial fatty acid beta-oxidation disorders (FAOD), are determined by a principal investigator or sub-investigator as beneficial to continue and also oneself wish to continue the clinical trial using bezafibrate. Secondarily to calculate the frequency of occurrence of muscle cramp attacks every week.
Basic objectives2 Safety
Basic objectives -Others
Trial characteristics_1 Confirmatory
Trial characteristics_2 Explanatory
Developmental phase Phase II,III

Assessment
Primary outcomes Safety endpoint
Existence and its contents of adverse events and side effects
Key secondary outcomes Efficacy endpoint
frequency of occurrence of muscle cramp attacks every week

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 Administer orally the following daily doses, which are equivalent to the end of the preceding study (HUPE-002-01), of the investigational drug twice a day after breakfast and dinner.

(1)for ages 3 and older, under 7.5;
Standard treatment period: 200mg (morning: 100mg, evening: 100mg)
When dose increased: 300mg (morning: 200mg, evening: 100mg)
(2)for ages 7.5 to 11;
Standard treatment period: 300mg (morning: 200mg, evening: 100mg)
When dose increased: 400mg (morning: 200mg, evening: 200mg)
(3)for ages 12 and older;
Standard treatment period: 600mg (morning: 300mg, evening: 300mg)
When dose increased: 800mg (morning: 400mg, evening: 400mg)
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
3 years-old <=
Age-upper limit
60 years-old >=
Gender Male and Female
Key inclusion criteria Patient who:
(1) was enrolled in the preceding study (HUPE-002-01) and completed Week 50
(2) gives written informed consent before enrolling the study.
The consent from a legally acceptable representative is required for the patients with uncertain capacity of judgments. Subjective patients under 20-year-old should receive an adequate explanation depending on one's ability to understand, and give assent with written forms if possible
Key exclusion criteria Patient who;
(1) is considered ineligible for enrolling the study by a principal investigator or sub-investigator
Target sample size 10

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Seiji Yamaguchi
Organization Shimane University Hospital
Division name Department of Pediatrics
Zip code
Address 89-1 En-ya-cho, Izumo, Shimane 693-8501, Japan
TEL 0853-20-2220
Email seijiyam@med.shimane-u.ac.jp

Public contact
Name of contact person
1st name
Middle name
Last name Saki Yokoshiki, Takashi Miyakoshi, Akiko Tamura
Organization Hokkaido University Hospital
Division name Clinical Research and Medical innovation Center Research and Development Division
Zip code
Address Kita14 Nishi5, Kita-ku, Sapporo, Hokkaido, 060-8648, Japan
TEL 011-706-7735
Homepage URL
Email Beza@pop.med.hokudai.ac.jp

Sponsor
Institute Shimane University Hospital
Institute
Department

Funding Source
Organization Clinical Trials Core Hospitals Project
Organization
Division
Category of Funding Organization
Nationality of Funding Organization Japan

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 北海道大学病院(北海道)、つがる西北五広域連合つがる総合病院(青森県)、日本大学病院(東京都)、岐阜大学医学部附属病院(岐阜県)、大阪大学医学部附属病院(大阪府)、市立八幡浜総合病院(愛媛県)、鹿児島市立病院(鹿児島県)、久留米大学病院(福岡県)、筑波大学附属病院(茨城県)

Other administrative information
Date of disclosure of the study information
2015 Year 04 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 Completed
Date of protocol fixation
2014 Year 08 Month 25 Day
Date of IRB
Anticipated trial start date
2015 Year 01 Month 01 Day
Last follow-up date
2017 Year 12 Month 01 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2015 Year 03 Month 27 Day
Last modified on
2015 Year 11 Month 24 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000019676

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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