Aerobic Training in Patients With Congenital Myopathies

Not Applicable

Completed

• Conditions: Congenital Myopathy
• Interventions: Other: Exercise on a cycle-ergometer

• Registration Number: NCT02020187
• Lead Sponsor: Rigshospitalet, Denmark

Brief Summary

Congenital myopathies are clinical and genetic heterogeneous disorders characterized by skeletal muscle weakness. Most patients experience muscle weakness and fatigue throughout their life. However, progression of symptoms is rare. There are no specific treatments for congenital myopathies. Training has been shown to benefit several other muscle diseases with weakness, but the defect in congenital myopathies involves contractile proteins of the sarcomere, why the effect of training is uncertain in these conditions. The investigators will therefore investigate the effect cycle-ergometer training for 30 minutes, three times weakly, for ten weeks in 15 patients with congenital myopathy. Another 5-10 congenital myopathy patients, who do not train, will serve as controls for the trained patients. The study starts and ends with a test day, where efficacy based on VO2max, performance in functional tests and a questionnaire will be assessed.

Detailed Description

10 weeks of cycle-ergometer training at home. Intensity at 70% of maximal VO2,max.

Study Timeline

• Study Start: 2013-09
• Study First Submitted: 2013-11-28
• Study First Submitted QC: 2013-12-18
• Study First Posted: 2013-12-24
• Primary Completion: 2014-11
• Study Completion: 2014-11
• Status Verified: 2015-05
• Last Update Submitted: 2015-05-11
• Last Update Posted: 2015-05-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

• Danish patients with gene verified congenital myopathy

Exclusion Criteria

• Patients who are to week to train at a cycle-ergometer for 10 weeks
• Patients who have other health issues, which can disturb the interpretation of the efficacy

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Exercise	Exercise on a cycle-ergometer	10 weeks of home training on a cycle-ergometer. Exercise 30 minutes every other day or at least three times a week.

Primary Outcome Measures

Name	Time	Method
Efficacy based on VO2,max	10 weeks	Difference in VO2,max from baseline to end of intervention.

Secondary Outcome Measures

Name	Time	Method
Dynamometry	10 weeks	Changes in external force production from baseline to after the intervention. Dynamometry measurements are done at hip flexion, dorsal foot flexion, plantar foot flexion, knee flexion, knee extension and elbow flexion.
Level of Creatine Kinase	10 weeks	Marker for exercise-induced muscle damage. Taken week 0, 3 and 10.
Intensity in maximal Watt	10 weeks	Changes in the maximal intensity (watt) from baseline VO2,max test to after the intervention.
6 minute walk test	10 weeks	Changes in the walking distance from baseline to after the intervention.
SF-36 questionnaire	10 weeks	Changes in SF-36 questionnaire from baseline to after the intervention.
A timed five-repetitions-sit-to-stand-test	10 weeks	Changes in time from baseline to after the intervention.
A timed 14-step-stair-test	10 weeks	Changes in time from baseline to after the intervention.

Trial Locations

Locations (1)

Neuromuscular Research Unit; 🇩🇰 Copenhagen, Denmark