FSHD - Facioscapulohumeral Muscular DystrophyFSHDNCT02159612
1 Year MRI Followup in Facioscapulohumeral Muscular Dystrophy
An observational study of FSHD - Facioscapulohumeral Muscular Dystrophy, run by Grete Andersen, MD at 1 site in Denmark.
Trial summary
An investigation of disease progression in adult danish patients with facioscapulohumeral muscular dystrophy. The disease progression is followed for a year with two test-days, including functional muscle tests and a MRI scan of muscles in the back and legs. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- FSHD - Facioscapulohumeral Muscular Dystrophy
- What is being tested?
- MRI
- How many people
- 45 participants
- Where
- 1 siteDenmark
- Study dates
- May 2014to Mar 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
- No healthy volunteers
- Adult, Older adult
Study population: Persons in Denmark diagnosed with Facioscapulohumeral muscular dystrophy is invited to the study.
Who may be able to join
- Diagnosed with FSHD type 1
Who cannot join
- Pregnant or breastfeeding women.
- Patients with claustrophobia.
- Patients with implanted metallic parts in the body.
Trial locations and contacts
1 site in 1 country.
Denmark1 site
Neuromuscular Research Unit, Department of Neurology, Rigshospitalet
Copenhagen, 2100
About this study
Official title: Disease Progression in Facioscapulohumeral Muscular Dystrophy - 1 Year MRI Follow-up
An investigation of disease progression in adult danish patients with facioscapulohumeral muscular dystrophy. The disease progression is followed for a year with two test-days, including functional muscle tests and a MRI scan of muscles in the back and legs.
Read the detailed description
A cohort of adult danish patients with facioscapulohumeral muscular dystrophy is invited to perform a MRI scan to describe the relation between backpain and disease affection in back muscles. After one year they are re-invited to perform another MRI scan to follow the disease progression.
The MRI scan is done to measure the quantity and quality of the muscles in the back, thigh and calf. Further, investigators investigate the muscle function by measuring of muscle strength in legs and in the back together with three functional tests. A walking test (6MWT), a stair test (14SST), and a chair test (5-TSTST). Self-assessment of back pain is measured by a standardized questionnaire about backpain.
- Being tested
- MRI
- Keywords
- FSHD, MRI, Disease progression
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral, Disease Progression
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MRIOther
MRI scan of muscles in the back and legs. Including T1, DIXON and STIR images.
| Study group | Role | What they receive |
|---|---|---|
| FSHD | A cohort of adult danish patients with facioscapulohumeral muscular dystrophy is invited to perform a MRI scan. Receives: Other: MRI |
What is being measured
The primary outcome is the main question the study is designed to answer.
Fat infiltration
Change in the distribution of fat infiltration in back, thigh and calves muscles seen at the MRI images.
Measured: 1 year
Secondary outcomes (4)
Muscle structure
Muscle structure is evaluated with a four-point semi-quantitative visual scale. Two observers evaluate images independently. Changes in evaluation between 1st and 2nd scan are the outcome measure.
Measured: 1 year
Muscle strength
Changes in muscle strength between 1st. and 2nd MRI-scan.
Measured: 1 year
Back pain
Changes in self-assessed back-pain between 1st. and 2nd scan.
Measured: 1 year
Muscle function
Changes in muscle function between 1st and 2nd MRI-scan, measured by a 6 minute walk test, a 14-step-stair test and a 5-times-sit-to-stand-test.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2014 (estimated)
- Study startMay 2014
- Primary completionMar 2015
- Study completionMar 2015
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 FSHD - Facioscapulohumeral Muscular DystrophyStudies of the same condition, open and under way.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Grete Andersen, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Grete Andersen, MD, Copenhagen University Hospital, Rigshospitalet
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02159612
- Sponsor's protocol ID
- H-3-2012-163 (fsh)
- Acronym
- FSHD
- Status last verified
- May 2015
- First submitted
- 6 Jun 2014
- Last updated
- 14 May 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Dahlqvist JR, Poulsen NS, Ostergaard ST, Fornander F, de Stricker Borch J, Danielsen ER, Thomsen C, Vissing J. Evaluation of inflammatory lesions over 2 years in facioscapulohumeral muscular dystrophy. Neurology. 2020 Sep 1;95(9):e1211-e1221. doi: 10.1212/WNL.0000000000010155. Epub 2020 Jul 1.
PubMed 32611642
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Disease Progression in Facioscapulohumeral Muscular Dystrophy - 1 Year MRI Follow-up
- ClinicalTrials.gov ID
- NCT02159612
- Acronym / sponsor ID
- FSHD / H-3-2012-163 (fsh)
- Keywords
- FSHD, MRI, Disease progression
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Grete Andersen, MDUniversity, hospital or other
- Study start
- May 2014
- Primary completion
- Mar 2015 (actual)
- Study completion
- Mar 2015 (actual)
- First posted
- 10 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.
An investigation of disease progression in adult danish patients with facioscapulohumeral muscular dystrophy. The disease progression is followed for a year with two test-days, including functional muscle tests and a MRI scan of muscles in the back and legs.
Detailed description
A cohort of adult danish patients with facioscapulohumeral muscular dystrophy is invited to perform a MRI scan to describe the relation between backpain and disease affection in back muscles. After one year they are re-invited to perform another MRI scan to follow the disease progression.
The MRI scan is done to measure the quantity and quality of the muscles in the back, thigh and calf. Further, investigators investigate the muscle function by measuring of muscle strength in legs and in the back together with three functional tests. A walking test (6MWT), a stair test (14SST), and a chair test (5-TSTST). Self-assessment of back pain is measured by a standardized questionnaire about backpain.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FSHD | – | Other: MRIA cohort of adult danish patients with facioscapulohumeral muscular dystrophy is invited to perform a MRI scan. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Persons in Denmark diagnosed with Facioscapulohumeral muscular dystrophy is invited to the study.
Key inclusion criteria (1 listed)
- Diagnosed with FSHD type 1
Key exclusion criteria (3 listed)
- Pregnant or breastfeeding women.
- Patients with claustrophobia.
- Patients with implanted metallic parts in the body.
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 |
|---|---|---|
| MRIMRI scan of muscles in the back and legs. Including T1, DIXON and STIR images. | Other | FSHD |
Primary outcome
Fat infiltration
Time frame: 1 year
Change in the distribution of fat infiltration in back, thigh and calves muscles seen at the MRI images.
4 secondary outcomes
Muscle structure
Time frame: 1 year
Muscle strength
Time frame: 1 year
Back pain
Time frame: 1 year
Muscle function
Time frame: 1 year
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 14 May 2015.
- Derived from this study: PubMed 32611642Dahlqvist JR, Poulsen NS, Ostergaard ST, Fornander F, de Stricker Borch J, Danielsen ER, Thomsen C, Vissing J. Evaluation of inflammatory lesions over 2 years in facioscapulohumeral muscular dystrophy. Neurology. 2020 Sep 1;95(9):e1211-e1221. doi: 10.1212/WNL.0000000000010155. Epub 2020 Jul 1.
- The home-page of neuromuscular research units department in Copenhagen, DenmarkLink provided by the sponsor.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 FSHD - Facioscapulohumeral Muscular Dystrophy, run by Grete Andersen, MD 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 3 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 14 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?
Fat infiltration (time frame: 1 year). 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 NCT02159612, 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 14 May 2015.