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

The multicenter continuous clinical trial of the safety and efficacy of bezafibrate for mitochondrial fatty acid beta-oxidation disorders - BezFAOD continuous study

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000016951

Enrollment

Registered

2015-04-01

Start date

2015-01-01

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

mitochondrial fatty acid beta-oxidation disorders

Interventions

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

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)

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 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

Exclusion criteria

Exclusion criteria: Patient who; (1) is considered ineligible for enrolling the study by a principal investigator or sub-investigator

Design outcomes

Primary

Measure	Time frame
Safety endpoint Existence and its contents of adverse events and side effects	—

Secondary

Measure	Time frame
Efficacy endpoint frequency of occurrence of muscle cramp attacks every week	—

Countries

Japan

Contacts

Public ContactSaki Yokoshiki, Takashi Miyakoshi, Akiko Tamura

Hokkaido University Hospital Clinical Research and Medical innovation Center Research and Development Division

Beza@pop.med.hokudai.ac.jp011-706-7735

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026