BezFAOD continuous clinical study

JRCT ID: jRCTs011180008

Registered date:05/03/2019

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: mitochondrial fatty acid beta-oxidation disorders
• Date of first enrollment: 19/08/2015
• Target sample size: 10
• Study type: Interventional
• Intervention(s): Oral administration of drug

Outcome(s)

Primary Outcome

Safety endpoint
Existence and its contents of adverse events and side effects

• Secondary Outcome: Measurement of QOL assessment (SF-36)

Key inclusion & exclusion criteria

• Age minimum: >= 3age old
• Age maximum: <= 60age old
• Gender: Both

Include 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

Exclude criteria

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

Related Information

• Primary Sponsor: shiraishi hideaki
• Secondary ID(s): UMIN000017984