Effect of Bezafibrate on muscle metabolism during exercise in patients with fat metabolic disorders in skeletal muscle - Bezafibrate study

Phase 1

• Conditions: Evaluation of the effect of Bezafibrate on muscle metabolism during exercise in patients with VLCAD and CPT II deficiency; MedDRA version: 12.0Level: LLTClassification code 10029316Term: Neuromuscular disorder NOS

• Registration Number: EUCTR2008-006704-27-DK
• Lead Sponsor: euromuscular Research Unit, Copenhagen University Hospital Rigshopspitalet

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2009-06-03
• Last Update Posted: 2 August 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 22

Inclusion Criteria

1.Significant decrease in the CPT II or VLCAD activities measured in lymphocytes or fibroblasts, together with genetic verification of either CPT II deficiency or VLCAD deficiency.
2.Males and females age 18-65 years.
3.All fertile females have to have a negative pregnancy test and use anti-conceptive during the study period.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Ingestion of competitive drugs, as described in the product resume of bezafibrate.
2.Competitive disorders, evaluated by the sponsor or investigators
3.Pregnancy or lactation during the period of fibrate therapy

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Secondary Objective: Secondary outcomes:<br>§Enzyme activity in leucocytes<br>§Acylcarnitine profile<br>§Accelerometer<br>§Borg score (perceived exertion)<br>§Number of episodes of myoglobinuria<br>§Daily muscle pain score (VAS)<br>§Daily diary of energy utilization (Bouchards questionnaire)<br>;Primary end point(s): The two primary outcomes to asses the potential effect of Bezafibrate is<br><br>§Fat oxidation and heart rate generated during physical activity on cycle ergometer at a constant workload<br><br>We expect the fat oxidation in the patients to increase by a factor 1.7.<br>;Main Objective: The two primary outcomes to asses the potential effect of Bezafibrate is<br><br>§Fat oxidation and heart rate generated during physical activity on cycle ergometer at a constant workload<br><br>We expect the fat oxidation in the patients to increase by a factor 1.7.<br>