Bethlem MyopathyNCT01895283
The Effect of Aerobic Exercise, on Fitness and Functional Muscle Strength, in Patients With Muscular Dystrophy
A study testing Moderate-intensity training in people with Bethlem Myopathy, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 to 90 yearsHealthy volunteers can take part
- Conditions the study is for
- Bethlem Myopathy
- What is being tested?
- Moderate-intensity training
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteDenmark
- Study dates
- Sep 2013to Jan 2015
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 90 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Patients with Bethlem myopathy
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Denmark1 site
Rigshospitalet, Neuromuscular Research Unit, section 3342
Copenhagen, 2100
About this study
Official title: The Effect of a Bike Ergometer Training Program in Patients With Bethlem Myopathy
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.
Read more
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.
- Conditions
- Bethlem Myopathy
- Being tested
- Moderate-intensity training
- Keywords
- Aerobic training, VO2max, Bethlem myopathy
- Medical subject headings
- Bethlem myopathy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Moderate-intensity trainingBehavioural
Training at 70% of VO2max, for 1/2 hour, three times weekly, for 10 weeks, on a bike ergometer.
| Study group | Role | What they receive |
|---|---|---|
| Moderate-intensity training | Experimental | Regular supervised moderate-intensity training on a bike ergometer, three times per week, for 30 minutes a total of 10 weeks. Receives: Behavioral: Moderate-intensity training |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximal oxygen uptake (VO2max)
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.
Measured: after 10 week bike ergometer training program
Secondary outcomes (4)
Functional muscle strength
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.
Measured: After completion of a 10 week training program
Muscle strength determined by dynamometry
Measured: After completion of a 10 week training program
Self-assessed improvements in muscle function
Measured: 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)
Measured: After a 10 week training program
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2013 (estimated)
- Study startSep 2013
- Primary completionDec 2014
- Study completionJan 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bethlem MyopathyStudies of the same condition, open and under way.
- More trials run by Copenhagen University Hospital, RigshospitaletThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Copenhagen University Hospital, Rigshospitalet
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Christoffer Rasmus Vissing, Copenhagen University Hospital, Rigshospitalet
- Investigators
- Christoffer R Vissing, BsC, Principal investigator, Rigshospitalet, Neuromuscular Research Unit
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01895283
- Sponsor's protocol ID
- H-2-2013-066
- Status last verified
- Apr 2016
- First submitted
- 3 Jul 2013
- Last updated
- 6 Apr 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of a Bike Ergometer Training Program in Patients With Bethlem Myopathy
- ClinicalTrials.gov ID
- NCT01895283
- Acronym / sponsor ID
- H-2-2013-066
- Conditions
- Bethlem Myopathy
- Keywords
- Aerobic training, VO2max, Bethlem myopathy
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Copenhagen University Hospital, RigshospitaletUniversity, hospital or other
- Study start
- Sep 2013
- Primary completion
- Dec 2014 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 10 Jul 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Moderate-intensity training | Experimental | Behavioral: Moderate-intensity trainingRegular supervised moderate-intensity training on a bike ergometer, three times per week, for 30 minutes a total of 10 weeks. |
Who may qualify
- Ages
- 16 to 90 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Patients with Bethlem myopathy
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Moderate-intensity trainingTraining at 70% of VO2max, for 1/2 hour, three times weekly, for 10 weeks, on a bike ergometer. | Behavioural | Moderate-intensity training |
Primary outcome
Maximal oxygen uptake (VO2max)
Time frame: 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.
4 secondary outcomes
Functional muscle strength
Time frame: After completion of a 10 week training program
Muscle strength determined by dynamometry
Time frame: After completion of a 10 week training program
Self-assessed improvements in muscle function
Time frame: 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)
Time frame: After a 10 week training program
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2016.
Related trials for Bethlem Myopathy
RecruitingObservational study
Congenital Muscle Disease Study of Patient and Family Reported Medical Information
Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christoffer R Vissing, BsC principal investigator
Rigshospitalet, Neuromuscular Research Unit
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Moderate-intensity training in people with Bethlem Myopathy, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Apr 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximal oxygen uptake (VO2max) (time frame: after 10 week bike ergometer training program). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Apr 2016.