BezFAOD continuous clinical study

Phase 2

• Conditions: mitochondrial fatty acid beta-oxidation disorders; fatty acid beta-oxidation disorders , Very-long-chain acyl-CoA dehydrogenase (VLCAD) deficiency, Carnitine palmitoyl transferase II deficiency, Glutaric acidaemia type 2

• Registration Number: JPRN-jRCTs011180008
• Lead Sponsor: shiraishi hideaki

Brief Summary

Regarding the QOL evaluation, which is the efficacy endpoint, improvement in the patient's QOL was observed. Regarding the safety endpoint of adverse events (AE) that occurred during the study period, no problematic AE were observed, and no side effects were observed, confirming the safety of daily bezafibrate administeration. In conclusion, in terms of QOL, the efficacy and safety of bezafibrate for FAOD were confirmed.

Detailed Description

Not available

Study Timeline

• Study Start: 2019-03-05
• Results First Posted: 2019-07-25
• Study Completion: 2022-05-09
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 7

Inclusion Criteria

Patient who
1.was enrolled in the preceding study HUPE-002-02 and completed Week 78
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 ones ability to understand, and give assent with written forms if possible

Exclusion Criteria

Patient who;
is considered ineligible for enrolling the study by a principal investigator or sub-investigator

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Safety endpoint <br>Existence and its contents of adverse events and side effects

Secondary Outcome Measures

Name	Time	Method
Measurement of QOL assessment (SF-36)