Bethlem Myopathy
Conditions
Keywords
Aerobic training, VO2max, Bethlem myopathy
Brief summary
Muscular dystrophy is a group of disorders that are characterized by progressive muscle weakening and loss of muscle mass, caused by defects in muscle proteins. Muscular dystrophy is almost always inherited disorders, and so far, no curative treatments exist. Previous studies have shown that endurance training significantly improves fitness and self-assessed muscle function in a variety of muscular dystrophies. In this study, we wish to investigate whether patients with Bethlem myopathy (a specific form of muscular dystrophy) also benefit from endurance training. The study consists of two test days, a 10-week training period and five blood tests. Patients will be required to train, three times per week, for 10 weeks, on a bike ergometer with a specific training intensity, under pulse rate monitoring. The training period will be flanked by two test days, where we will determine, and compare the patients' muscle strength and fitness level, from before to after the training program. The patients conditioning level will be determined from a 15 minute cycle ergometer test and patients will undergo three functional tests to determine their functional muscle strength. We will as a safety measure analyze blood tests before, during and after the training program for the muscle enzyme creatine kinase (an indicator of muscle damage) and through adverse effects as reported by patients during weekly telephone-consultations with the Principal investigator. We anticipate, that Bethlem myopathy patients will have a similar rise in fitness level and functional muscle strength, as that seen in patients with other forms of muscular dystrophy, who undertake a similar training program.
Interventions
BEHAVIORALModerate-intensity training
Training at 70% of VO2max, for 1/2 hour, three times weekly, for 10 weeks, on a bike ergometer.
Sponsors
Rigshospitalet, Denmark
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
16 Years to 90 Years
Healthy volunteers
Yes
Inclusion criteria
* Patients with Bethlem myopathy
Exclusion criteria
* Patients with disorders that would confound the interpretation of the effect from training (cardiomyopathy, pregnant women, etc.) * Patients who are deemed mentally unfit for cooperation with the training program
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Maximal oxygen uptake (VO2max) | after 10 week bike ergometer training program | VO2max will be determined during an incremental bike test to exhaustion. VO2max is a physiological measure of physical fitness. VO2max relies on a persons ability to supply and consume oxygen (energy) in muscle tissue. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Functional muscle strength | After completion of a 10 week training program | Functional muscle strength will be determined by performance in three functional tests: a five-repetition-sit-to-stand-test, a 6 minute walk test and a 14 step stair test. |
| Muscle strength determined by dynamometry | After completion of a 10 week training program | — |
| Self-assessed improvements in muscle function | After completion of a 10 week training program | — |
| Self-assessed improvements in incidence of problems that are related to inactivity (in patients who are confined to a wheel-chair) | After a 10 week training program | — |
Other
| Measure | Time frame | Description |
|---|---|---|
| Safety | During a 10 week training program | The safety of undertaking a 10-week training program will be determined by analyzing reports of adverse effects faced during the training program, and by interpretation of Creatine Kinase levels measured before, during and after the training program. |
Countries
Denmark
Outcome results
None listed