1 Year MRI Followup in Facioscapulohumeral Muscular Dystrophy

Completed

• Conditions: FSHD - Facioscapulohumeral Muscular Dystrophy
• Interventions: Other: MRI

• Registration Number: NCT02159612
• Lead Sponsor: Grete Andersen, MD

Brief 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.

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 Timeline

• Study Start: 2014-05
• Study First Submitted: 2014-06-06
• Study First Submitted QC: 2014-06-06
• Study First Posted: 2014-06-10
• Primary Completion: 2015-03
• Study Completion: 2015-03
• Status Verified: 2015-05
• Last Update Submitted: 2015-05-13
• Last Update Posted: 2015-05-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 45

Inclusion Criteria

• Diagnosed with FSHD type 1

Exclusion Criteria

• Pregnant or breastfeeding women.
• Patients with claustrophobia.
• Patients with implanted metallic parts in the body.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
FSHD	MRI	A cohort of adult danish patients with facioscapulohumeral muscular dystrophy is invited to perform a MRI scan.

Primary Outcome Measures

Name	Time	Method
Fat infiltration	1 year	Change in the distribution of fat infiltration in back, thigh and calves muscles seen at the MRI images.

Secondary Outcome Measures

Name	Time	Method
Back pain	1 year	Changes in self-assessed back-pain between 1st. and 2nd scan.
Muscle strength	1 year	Changes in muscle strength between 1st. and 2nd MRI-scan.
Muscle structure	1 year	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.
Muscle function	1 year	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.

Trial Locations

Locations (1)

Neuromuscular Research Unit, Department of Neurology, Rigshospitalet; 🇩🇰 Copenhagen, Denmark