Unique ID issued by UMIN | UMIN000016951 |
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Receipt number | 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 22:07:20 |
The multicenter continuous clinical trial of the safety and efficacy of bezafibrate for mitochondrial fatty acid beta-oxidation disorders
BezFAOD continuous study
The multicenter continuous clinical trial of the safety and efficacy of bezafibrate for mitochondrial fatty acid beta-oxidation disorders
BezFAOD continuous study
Japan |
mitochondrial fatty acid beta-oxidation disorders
Pediatrics |
Others
NO
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.
Safety
Confirmatory
Explanatory
Phase II,III
Safety endpoint
Existence and its contents of adverse events and side effects
Efficacy endpoint
frequency of occurrence of muscle cramp attacks every week
Interventional
Single arm
Non-randomized
Open -no one is blinded
Uncontrolled
1
Treatment
Medicine |
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)
3 | years-old | <= |
60 | years-old | >= |
Male and Female
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
Patient who;
(1) is considered ineligible for enrolling the study by a principal investigator or sub-investigator
10
1st name | |
Middle name | |
Last name | Seiji Yamaguchi |
Shimane University Hospital
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 | Saki Yokoshiki, Takashi Miyakoshi, Akiko Tamura |
Hokkaido University Hospital
Clinical Research and Medical innovation Center Research and Development Division
Kita14 Nishi5, Kita-ku, Sapporo, Hokkaido, 060-8648, Japan
011-706-7735
Beza@pop.med.hokudai.ac.jp
Shimane University Hospital
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)
2015 | Year | 04 | Month | 01 | Day |
Unpublished
Completed
2014 | Year | 08 | Month | 25 | Day |
2015 | Year | 01 | Month | 01 | Day |
2017 | Year | 12 | Month | 01 | Day |
2015 | Year | 03 | Month | 27 | Day |
2015 | Year | 11 | Month | 24 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000019676
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