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A Study of Bezafibrate in Mitochondrial Myopathy

NCT02398201 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2017-09-21

No results posted yet for this study

Summary

The purpose of this study is to gather preliminary data on whether bezafibrate can improve cellular energy production in mitochondrial disease.

Mitochondrial diseases are rare inherited disorders that arise due to deficient energy production within the cells of the body. Consequently, the typical clinical features arise in organs with high energy requirements. Mitochondrial disorders exhibit highly variable clinical effects, both between individuals and within families. Characteristic symptoms include muscle weakness (myopathy), hearing loss, migraine, epilepsy and stroke like episodes in addition to diabetes and heart problems. Mitochondrial disorders can therefore impact considerably on both quality of life and life expectancy. Despite this, no proven disease modifying treatments are available.

Pre-clinical studies have identified that several existing medications improve mitochondrial function. Of these, bezafibrate has the best supportive data and, because it is already licensed as a treatment for high blood fats, has a well characterised side effect profile.

The investigators will therefore conduct a feasibility study of bezafibrate in people with mitochondrial myopathy. Ten affected participants will be recruited and will receive a titrating course of bezafibrate three times daily for 12 weeks.

Conditions

  • Mitochondrial Diseases

Interventions

DRUG

Bezafibrate

Bezafibrate 200mg-600mg three times daily for 12 weeks.

Sponsors & Collaborators

  • Newcastle University

    collaborator OTHER

  • Newcastle-upon-Tyne Hospitals NHS Trust

    lead OTHER

Principal Investigators

  • Patrick F Chinnery, MBBS, PhD · Newcastle Univeristy

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
64 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-09-30
Primary Completion
2017-01-31
Completion
2017-03-23

Countries

  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02398201 on ClinicalTrials.gov