Aerobic Training in Patients With Congenital Myopathies
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.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Supportive Care
|Official Title:||Aerobic Training in Patients With Congenital Myopathies|
- Efficacy based on VO2,max [ Time Frame: 10 weeks ] [ Designated as safety issue: No ]Difference in VO2,max from baseline to end of intervention.
- Level of Creatine Kinase [ Time Frame: 10 weeks ] [ Designated as safety issue: Yes ]Marker for exercise-induced muscle damage. Taken week 0, 3 and 10.
- Intensity in maximal Watt [ Time Frame: 10 weeks ] [ Designated as safety issue: No ]Changes in the maximal intensity (watt) from baseline VO2,max test to after the intervention.
- 6 minute walk test [ Time Frame: 10 weeks ] [ Designated as safety issue: No ]Changes in the walking distance from baseline to after the intervention.
- SF-36 questionnaire [ Time Frame: 10 weeks ] [ Designated as safety issue: No ]Changes in SF-36 questionnaire from baseline to after the intervention.
- A timed five-repetitions-sit-to-stand-test [ Time Frame: 10 weeks ] [ Designated as safety issue: No ]Changes in time from baseline to after the intervention.
- A timed 14-step-stair-test [ Time Frame: 10 weeks ] [ Designated as safety issue: No ]Changes in time from baseline to after the intervention.
- Dynamometry [ Time Frame: 10 weeks ] [ Designated as safety issue: No ]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.
|Study Start Date:||September 2013|
|Estimated Study Completion Date:||November 2014|
|Estimated Primary Completion Date:||November 2014 (Final data collection date for primary outcome measure)|
10 weeks of home training on a cycle-ergometer. Exercise 30 minutes every other day or at least three times a week.
Other: Exercise on a cycle-ergometer
Home training on an ergometer-cycle 30 minutes every other day or at least three times a week. Subject will exercise in a pulse interval corresponding to 70% of their maximal VO2.
No Intervention: Controls
Controls with diagnosed congenital myopathy. Subjects are tested two times on a cycle ergometer. There will be ten weeks between the tests. In between tests the subjects are living life as usual without any interventions.
10 weeks of cycle-ergometer training at home. Intensity at 70% of maximal VO2,max.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02020187
|Neuromuscular Research Unit||Recruiting|
|Copenhagen, Denmark, 2100|
|Contact: Vissing +4535451842|
|Principal Investigator:||Gitte H Pedersen, Bsc Medicine||Rigshospitalet, Denmark|