mitochondrial fatty acid Beta-oxidation disorders
Conditions
Interventions
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 1
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
Run-in period: 400mg (morning: 200mg, evening: 200mg)
Standard treatment period: 600mg (morning: 300mg, evening: 300mg)
When dose increased: 800mg (morning: 400mg, evening: 400mg)
Sponsors
Shimane University School of Medicine
Eligibility
Sex/Gender
All
Inclusion criteria
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.
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Measurement of muscle cramp attacks every week before and after Bezafibrate treatment. | — |
Secondary
| Measure | Time frame |
|---|---|
| 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. | — |
Countries
Japan
Outcome results
None listed