Congenital MyopathyNCT02020187
Aerobic Training in Patients With Congenital Myopathies
A study testing Exercise on a cycle-ergometer in people with Congenital Myopathy, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Congenital Myopathy
- What is being tested?
- Exercise on a cycle-ergometer
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteDenmark
- Study dates
- Sep 2013to Nov 2014
- 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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Danish patients with gene verified congenital myopathy
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the detailed description
10 weeks of cycle-ergometer training at home. Intensity at 70% of maximal VO2,max.
- Conditions
- Congenital Myopathy
- Being tested
- Exercise on a cycle-ergometer
- Keywords
- Congenital myopathy, Exercise
- Medical subject headings
- Myotonia Congenita, Motor Activity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Exercise on a cycle-ergometerOther
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.
| Study group | Role | What they receive |
|---|---|---|
| Exercise | Experimental | 10 weeks of home training on a cycle-ergometer. Exercise 30 minutes every other day or at least three times a week. Receives: 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy based on VO2,max
Difference in VO2,max from baseline to end of intervention.
Measured: 10 weeks
Secondary outcomes (7)
Level of Creatine Kinase
Marker for exercise-induced muscle damage. Taken week 0, 3 and 10.
Measured: 10 weeks
Intensity in maximal Watt
Changes in the maximal intensity (watt) from baseline VO2,max test to after the intervention.
Measured: 10 weeks
6 minute walk test
Changes in the walking distance from baseline to after the intervention.
Measured: 10 weeks
SF-36 questionnaire
Changes in SF-36 questionnaire from baseline to after the intervention.
Measured: 10 weeks
A timed five-repetitions-sit-to-stand-test
Changes in time from baseline to after the intervention.
Measured: 10 weeks
A timed 14-step-stair-test
Changes in time from baseline to after the intervention.
Measured: 10 weeks
Dynamometry
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.
Measured: 10 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2013 (estimated)
- Study startSep 2013
- Primary completionNov 2014
- Study completionNov 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Congenital 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
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Copenhagen University Hospital, Rigshospitalet
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Gitte Hedermann Pedersen, Copenhagen University Hospital, Rigshospitalet
- Investigators
- Gitte H Pedersen, Bsc Medicine, Principal investigator, Copenhagen University Hospital, Rigshospitalet
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02020187
- Sponsor's protocol ID
- H2-2013-066
- Status last verified
- May 2015
- First submitted
- 28 Nov 2013
- Last updated
- 12 May 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 26751952
Study at a glance
From the registry record, last updated by the sponsor on 12 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Aerobic Training in Patients With Congenital Myopathies
- ClinicalTrials.gov ID
- NCT02020187
- Acronym / sponsor ID
- H2-2013-066
- Conditions
- Congenital Myopathy
- Keywords
- Congenital myopathy, Exercise
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Copenhagen University Hospital, RigshospitaletUniversity, hospital or other
- Study start
- Sep 2013
- Primary completion
- Nov 2014 (actual)
- Study completion
- Nov 2014 (actual)
- First posted
- 24 Dec 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.
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.
Detailed description
10 weeks of cycle-ergometer training at home. Intensity at 70% of maximal VO2,max.
Study design
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Exercise | Experimental | Other: Exercise on a cycle-ergometer10 weeks of home training on a cycle-ergometer. Exercise 30 minutes every other day or at least three times a week. |
| 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. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Danish patients with gene verified congenital myopathy
Key exclusion criteria (2 listed)
- 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
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 |
|---|---|---|
| Exercise on a cycle-ergometerHome 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. | Other | Exercise |
Primary outcome
Efficacy based on VO2,max
Time frame: 10 weeks
Difference in VO2,max from baseline to end of intervention.
7 secondary outcomes
Level of Creatine Kinase
Time frame: 10 weeks
Intensity in maximal Watt
Time frame: 10 weeks
6 minute walk test
Time frame: 10 weeks
SF-36 questionnaire
Time frame: 10 weeks
A timed five-repetitions-sit-to-stand-test
Time frame: 10 weeks
A timed 14-step-stair-test
Time frame: 10 weeks
Dynamometry
Time frame: 10 weeks
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 12 May 2015.
- Derived from this study: PubMed 26751952Hedermann 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.
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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.
Gitte H Pedersen, Bsc Medicine principal investigator
Copenhagen University Hospital, Rigshospitalet
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Exercise on a cycle-ergometer in people with Congenital 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 12 May 2015. 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?
Efficacy based on VO2,max (time frame: 10 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02020187, which also lists 1 linked publication.
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 12 May 2015.