Becker Muscular DystrophyNCT02165358
Muscle MRI in Becker Muscular Dystrophy and in Limb-girdle Muscular Dystrophy Type 2I
An observational study of Becker Muscular Dystrophy and Limb-Girdle Muscular Dystrophy Type 2I, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.
Trial summary
The purpose of this study is to investigate the paradoxical muscle enlargement in the calves and tongue seen in patients affected by Becker muscular dystrophy and Limb-girdle muscular dystrophy type 2I. Read more about this study
- Who is it for?
- Ages 18 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2I
- How many people
- 38 participants
- Where
- 1 siteDenmark
- Study dates
- Jun 2014to Jan 2015
- Phase
- Observational
- 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 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Danish patients genetically verified with either becker muscular dystrophy or limb-girdle muscular dystrophy type 2I
Who may be able to join
- Danish patients genetically verified with either becker muscular dystrophy or limb-girdle muscular dystrophy type 2I
- calves enlargement
Who cannot join
- Patients who have metal implants in the body, for instance surgical clips, cochlear implants, pacemaker etc.
- pregnant or breast-feeding patients
- patients who are claustrophobic
- atrophic calves
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Muscle MRI Study of Patients With Becker Muscular Dystrophy and Limb-girdle Muscular Dystrophy Type 2I
The purpose of this study is to investigate the paradoxical muscle enlargement in the calves and tongue seen in patients affected by Becker muscular dystrophy and Limb-girdle muscular dystrophy type 2I.
The enlarged calves' muscle quality will be assessed primarily on the basis of the muscle structure on MRI and based on a calculation of muscle strength per cross-sectional area.The findings will be compared with results from non-affected controls.
Additionally we want to describe the tongue muscle appearance on T1-weighted MRI.
Read the detailed description
The purpose of this study is to investigate the paradoxical muscle enlargement seen in patients affected by Becker muscular dystrophy and Limb-girdle muscular dystrophy type 2I.
Becker and limb-girdle muscular dystrophies are both hereditary muscular disorders. Both diseases appear clinically with shrinkage and muscle weakness of the muscles around the shoulder girdle and pelvis, while muscle hypertrophy of the calves and tongue is seen.
The quality of the enlarged calves has to the investigators knowledge not previously been described, and their hypothesis is that the normal correlation between muscle strength and muscle cross-section is absent in the calf muscles. Thus the investigators hypothesis suggests that there is no actual hypertrophy of the calf muscles, but rather a pseudohypertrofy with reduced force per cross-sectional area. With a picture of increased fat infiltration and disorganized muscle fibers.
The enlarged calves' muscle quality will be assessed primarily on the basis of the muscle structure on MRI (assessed on both T1 imaging, 3-point Dixon scans, and diffusion tension imaging), and based on a calculation of muscle strength per cross-sectional area. Isokinetic muscle dynamometry (Biodex-4) will be used to measure muscle strength. The findings will be compared with results from non-affected controls.
Additionally the investigators want to describe the tongue muscle appearance on T1-weighted MRI.
- Keywords
- BMD, LGMD2I, Calf enlargement, Muscular dystrophy, MRI
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophy, Limb-Girdle, Type 2I, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Patients | Patients verified with either becker muscular dystrophy or limb-girdle muscular dystrophy type 2I | |
| Controls | Healthy controls matched for age and gender. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Calves muscle quality
the investigators will describe muscle quality by:
- MRI scans of the calves comprising of a T1-weighted MRI scan, a 3-point Dixon scan to measure the fraction of fat in the muscle, a short TI inversion recovery (STIR) scan, and a diffusion tensor imaging (DTI) scan to see the muscle fiber organization. The MRI scans will further be used to measure the fat free muscle cross-sectional area of the muscle groups responsible for plantar flexion and dorsi flexion respectively.
- Biodex muscle strength measurements: Isokinetic muscle dynamometry measurements on plantar flexion and dorsal flexion over the ankle.
- Calculating of muscle strength per muscle cross-sectional area
Measured: 1 day
Secondary outcomes (1)
MRI T1 scan of the tongue
MRI T1 scan of the tongue to investigate muscle volume
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2014 (estimated)
- Study startJun 2014
- Primary completionJan 2015
- Study completionJan 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Becker Muscular DystrophyStudies 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
- Observational
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- Copenhagen University Hospital, Rigshospitalet
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Nicoline Løkken, Copenhagen University Hospital, Rigshospitalet
- Investigators
- Nicoline Løkken, Bsc, Principal investigator, Neuromuscular Research Unit, Rigshospitalet, Copenhagen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02165358
- Sponsor's protocol ID
- H-2-2014-035 (MRI BMD/LGMD2I)
- Status last verified
- Nov 2016
- First submitted
- 11 Jun 2014
- Last updated
- 11 Nov 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Muscle MRI Study of Patients With Becker Muscular Dystrophy and Limb-girdle Muscular Dystrophy Type 2I
- ClinicalTrials.gov ID
- NCT02165358
- Acronym / sponsor ID
- H-2-2014-035 (MRI BMD/LGMD2I)
- Keywords
- BMD, LGMD2I, Calf enlargement, Muscular dystrophy, MRI
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- Copenhagen University Hospital, RigshospitaletUniversity, hospital or other
- Study start
- Jun 2014
- Primary completion
- Jan 2015 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 17 Jun 2014 (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.
The purpose of this study is to investigate the paradoxical muscle enlargement in the calves and tongue seen in patients affected by Becker muscular dystrophy and Limb-girdle muscular dystrophy type 2I.
The enlarged calves' muscle quality will be assessed primarily on the basis of the muscle structure on MRI and based on a calculation of muscle strength per cross-sectional area.The findings will be compared with results from non-affected controls.
Additionally we want to describe the tongue muscle appearance on T1-weighted MRI.
Detailed description
The purpose of this study is to investigate the paradoxical muscle enlargement seen in patients affected by Becker muscular dystrophy and Limb-girdle muscular dystrophy type 2I.
Becker and limb-girdle muscular dystrophies are both hereditary muscular disorders. Both diseases appear clinically with shrinkage and muscle weakness of the muscles around the shoulder girdle and pelvis, while muscle hypertrophy of the calves and tongue is seen.
The quality of the enlarged calves has to the investigators knowledge not previously been described, and their hypothesis is that the normal correlation between muscle strength and muscle cross-section is absent in the calf muscles. Thus the investigators hypothesis suggests that there is no actual hypertrophy of the calf muscles, but rather a pseudohypertrofy with reduced force per cross-sectional area. With a picture of increased fat infiltration and disorganized muscle fibers.
The enlarged calves' muscle quality will be assessed primarily on the basis of the muscle structure on MRI (assessed on both T1 imaging, 3-point Dixon scans, and diffusion tension imaging), and based on a calculation of muscle strength per cross-sectional area. Isokinetic muscle dynamometry (Biodex-4) will be used to measure muscle strength. The findings will be compared with results from non-affected controls.
Additionally the investigators want to describe the tongue muscle appearance on T1-weighted MRI.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients | – | –Patients verified with either becker muscular dystrophy or limb-girdle muscular dystrophy type 2I |
| Controls | – | –Healthy controls matched for age and gender. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Danish patients genetically verified with either becker muscular dystrophy or limb-girdle muscular dystrophy type 2I
Key inclusion criteria (2 listed)
- Danish patients genetically verified with either becker muscular dystrophy or limb-girdle muscular dystrophy type 2I
- calves enlargement
Key exclusion criteria (4 listed)
- Patients who have metal implants in the body, for instance surgical clips, cochlear implants, pacemaker etc.
- pregnant or breast-feeding patients
- patients who are claustrophobic
- atrophic calves
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Calves muscle quality
Time frame: 1 day
the investigators will describe muscle quality by: 1. MRI scans of the calves comprising of a T1-weighted MRI scan, a 3-point Dixon scan to measure the fraction of fat in the muscle, a short TI inversion recovery (STIR) scan, and a diffusion tensor imaging (DTI) scan to see the muscle fiber organization. The MRI scans will further be used to measure the fat free muscle cross-sectional area of the muscle groups responsible for plantar flexion and dorsi flexion respectively. 2. Biodex muscle strength measurements: Isokinetic muscle dynamometry measurements on plantar flexion and dorsal flexion over the ankle. 3. Calculating of muscle strength per muscle cross-sectional area
1 secondary outcome
MRI T1 scan of the tongue
Time frame: 1 day
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 11 Nov 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.
Nicoline Løkken, Bsc principal investigator
Neuromuscular Research Unit, Rigshospitalet, Copenhagen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Becker Muscular Dystrophy and Limb-Girdle Muscular Dystrophy Type 2I, 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 2 inclusion and 4 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 11 Nov 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?
Calves muscle quality (time frame: 1 day). 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 11 Nov 2016.