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

Phase 2

• Conditions: mitochondrial fatty acid Beta-oxidation disorders

• Registration Number: JPRN-UMIN000012934
• Lead Sponsor: Shimane University School of Medicine

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-01-24
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Complete: follow-up complete
• Sex: All
• Target Recruitment: 10

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

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

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

Secondary Outcome Measures

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