Unique ID issued by UMIN | UMIN000012934 |
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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 |
The multicenter clinical trial of the efficacy and safety of bezafibrate for mitochondrial fatty acid Beta-oxidation disorders
BezFAOD study
The multicenter clinical trial of the efficacy and safety of bezafibrate for mitochondrial fatty acid Beta-oxidation disorders
BezFAOD study
Japan |
mitochondrial fatty acid Beta-oxidation disorders
Pediatrics |
Others
NO
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.
Efficacy
Confirmatory
Explanatory
Phase II,III
Measurement of muscle cramp attacks every week before and after Bezafibrate treatment.
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.
Interventional
Single arm
Non-randomized
Open -no one is blinded
Uncontrolled
1
Treatment
Medicine |
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)
3 | years-old | <= |
60 | years-old | >= |
Male and Female
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.
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
10
1st name | |
Middle name | |
Last name | Seiji Yamaguchi |
Shimane University School of Medicine
Department of Pediatrics
89-1 En-ya-cho, Izumo, Shimane 693-8501, Japan
0853-20-2220
seijiyam@med.shimane-u.ac.jp
1st name | |
Middle name | |
Last name | Tsuyoshi Teramoto, Takashi Miyakoshi, Ayako Hirai, Akiko Tamura |
Hokkaido University Hospital
Translational Research and Clinical Trial Center Clinical Research Strategic Division
Kita14 Nishi5, Kita-ku, Sapporo, Hokkaido, 060-8648, Japan
011-706-7735
Beza@pop.med.hokudai.ac.jp
Shimane University School of Medicine
Clinical Trials Core Hospitals Project
Japan
NO
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)
2014 | Year | 01 | Month | 24 | Day |
Unpublished
Completed
2013 | Year | 11 | Month | 12 | Day |
2014 | Year | 01 | Month | 30 | Day |
2016 | Year | 03 | Month | 30 | Day |
2014 | Year | 01 | Month | 23 | Day |
2015 | Year | 11 | Month | 24 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000015101
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