Aerobic Training in Patients With Congenital Myopathies

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2014 by Rigshospitalet, Denmark
Sponsor:
Information provided by (Responsible Party):
Gitte Hedermann Pedersen, Rigshospitalet, Denmark
ClinicalTrials.gov Identifier:
NCT02020187
First received: November 28, 2013
Last updated: June 6, 2014
Last verified: June 2014
  Purpose

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.


Condition Intervention
Congenital Myopathy
Other: Exercise on a cycle-ergometer

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by Rigshospitalet, Denmark:

Primary Outcome Measures:
  • Efficacy based on VO2,max [ Time Frame: 10 weeks ] [ Designated as safety issue: No ]
    Difference in VO2,max from baseline to end of intervention.


Secondary Outcome Measures:
  • 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.


Estimated Enrollment: 25
Study Start Date: September 2013
Estimated Study Completion Date: October 2014
Estimated Primary Completion Date: July 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Exercise
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.

Detailed Description:

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

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02020187

Locations
Denmark
Neuromuscular Research Unit Recruiting
Copenhagen, Denmark, 2100
Contact: Vissing    +4535451842      
Sponsors and Collaborators
Rigshospitalet, Denmark
Investigators
Principal Investigator: Gitte H Pedersen, Bsc Medicine Rigshospitalet, Denmark
  More Information

No publications provided

Responsible Party: Gitte Hedermann Pedersen, Medical student, Bsc, Rigshospitalet, Denmark
ClinicalTrials.gov Identifier: NCT02020187     History of Changes
Other Study ID Numbers: H2-2013-066
Study First Received: November 28, 2013
Last Updated: June 6, 2014
Health Authority: Denmark: Ethics Committee
Denmark: Danish Dataprotection Agency

Keywords provided by Rigshospitalet, Denmark:
Congenital myopathy
Exercise

Additional relevant MeSH terms:
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases

ClinicalTrials.gov processed this record on August 20, 2014