UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000012934
Receipt number R000015101
Scientific Title The multicenter clinical trial of the efficacy and safety of bezafibrate for mitochondrial fatty acid Beta-oxidation disorders
Date of disclosure of the study information 2014/01/24
Last modified on 2015/11/24 22:06:31

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Basic information

Public title

The multicenter clinical trial of the efficacy and safety of bezafibrate for mitochondrial fatty acid Beta-oxidation disorders

Acronym

BezFAOD study

Scientific Title

The multicenter clinical trial of the efficacy and safety of bezafibrate for mitochondrial fatty acid Beta-oxidation disorders

Scientific Title:Acronym

BezFAOD 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

Evaluation of the efficacy and safety of BF-759 (Bezafibrate) for patients with mitochondrial fatty acid Beta-oxidation disorders (FAOD) by the multicenter uncontrolled open-label trial.

Basic objectives2

Efficacy

Basic objectives -Others


Trial characteristics_1

Confirmatory

Trial characteristics_2

Explanatory

Developmental phase

Phase II,III


Assessment

Primary outcomes

Measurement of muscle cramp attacks every week before and after Bezafibrate treatment.

Key secondary outcomes

Measurement of the AC values during attacks before and after Bezafibrate treatment.
Measurement of the CK values during attacks before and after Bezafibrate treatment.
Measurement of VAS during attacks before and after Bezafibrate treatment.
Measurement of QOL assessment (SF-36) at the beginning and end of the trial.


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 of the investigational drug twice a day after breakfast and dinner.

(1) for ages 3 and older, under 7.5;
Run-in period: 100mg (morning: 100mg, evening: none)
Standard treatment period: 200mg (morning: 100mg, evening: 100mg)
When dose increased: 300mg (morning: 200mg, evening: 100mg)
(2) for ages 7.5 to 11;
Run-in period: 200mg (morning: 100mg, evening: 100mg)
Standard treatment period: 300mg (morning: 200mg, evening: 100mg)
When dose increased: 400mg (morning: 200mg, evening: 200mg)
(3) for ages 12 and older;
Run-in period: 400mg (morning: 200mg, evening: 200mg)
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) ages 3 to 60 at the time of obtaining informed consent.
(2) is enzymologically or genetically-proven as FAOD (any oneof CPT II, VLCAD, TFP, GA2, CPT I, MCAD, CACT) at the time of obtaining informed consent.
(3) weighs over 10.0kg at the time of screening test.
(4) has had episodes of muscular symptoms (general malaise, strong stiff shoulder or back pain, myalgia, lassitude of extremities, muscle weakness, myoglobinuria or subject-specific muscular symptoms) twice and more within 6 months or 4 times and more within 1 year prior to obtaining informed consent.
(5) has had the CK value which was more than double ULN at the time of attacks within 3 months prior to obtaining informed consent.
(6) 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) has the serum creatinine level more than 1.5 mg/dL at the time of screening test
(2) has a heart disease or liver damage requiring treatment
(3) has gallstones or a previous history of them
(4) is treated with HMG-CoA reductase inhibitor
(5) takes Bezafibrate or have a history of taking it
(6) is pregnant or nursing women, or expected to (wishing and planning to) become pregnant
(7) is considered ineligible for enrolling the study by a principal investigator or subinvestigator

Target sample size

10


Research contact person

Name of lead principal investigator

1st name
Middle name
Last name Seiji Yamaguchi

Organization

Shimane University School of Medicine

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 Tsuyoshi Teramoto, Takashi Miyakoshi, Ayako Hirai, Akiko Tamura

Organization

Hokkaido University Hospital

Division name

Translational Research and Clinical Trial Center Clinical Research Strategic 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 or person

Institute

Shimane University School of Medicine

Institute

Department

Personal name



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西北中央病院(青森県)、駿河台日本大学病院(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)


Other administrative information

Date of disclosure of the study information

2014 Year 01 Month 24 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

2013 Year 11 Month 12 Day

Date of IRB


Anticipated trial start date

2014 Year 01 Month 30 Day

Last follow-up date

2016 Year 03 Month 30 Day

Date of closure to data entry


Date trial data considered complete


Date analysis concluded



Other

Other related information



Management information

Registered date

2014 Year 01 Month 23 Day

Last modified on

2015 Year 11 Month 24 Day



Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000015101


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name