Aerobic Training in Patients With Congenital Myopathies

NCT02020187 | Completed

• 1 other identifier: H2-2013-066
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

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.

Conditions

Congenital Myopathy

Keywords

Congenital myopathy; Exercise

Outcome Measures

Primary Outcomes (1)

• Efficacy based on VO2,max

  Difference in VO2,max from baseline to end of intervention.

  10 weeks

Secondary Outcomes (7)

• Level of Creatine Kinase

  10 weeks

• Intensity in maximal Watt

  10 weeks

• 6 minute walk test

  10 weeks

• SF-36 questionnaire

  10 weeks

• A timed five-repetitions-sit-to-stand-test

  10 weeks

Study Arms (2)

Exercise

EXPERIMENTAL

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

Controls

NO INTERVENTION

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.

Interventions

Exercise on a cycle-ergometer OTHER

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.

Exercise

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Danish patients with gene verified congenital myopathy

You may not qualify if:

• 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

Sponsors & Collaborators

• Rigshospitalet, Denmark: lead

Study Sites (1)

Neuromuscular Research Unit

Copenhagen, 2100, Denmark

Location

Related Publications (1)

• Hedermann G, Vissing CR, Heje K, Preisler N, Witting N, Vissing J. Aerobic Training in Patients with Congenital Myopathy. PLoS One. 2016 Jan 11;11(1):e0146036. doi: 10.1371/journal.pone.0146036. eCollection 2016.

  PMID: 26751952 DERIVED

MeSH Terms

Conditions

Myotonia Congenita; Motor Activity

Condition Hierarchy (Ancestors)

Myotonic Disorders; Muscular Diseases; Musculoskeletal Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Behavior

Study Officials

• Gitte H Pedersen, Bsc Medicine

  Rigshospitalet, Denmark

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical student, Bsc

Study Record Dates

• First Submitted: November 28, 2013
• First Posted: December 24, 2013
• Study Start: September 1, 2013
• Primary Completion: November 1, 2014
• Study Completion: November 1, 2014
• Last Updated: May 12, 2015

Record last verified: 2015-05

Locations

DK (1)