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Clinical Trials/NCT03693898
NCT03693898
Unknown
Not Applicable

MR in Patients With Neuromuscular Diseases

Ruth Salim1 site in 1 country20 target enrollmentSeptember 1, 2018

Overview

Phase
Not Applicable
Intervention
Not specified
Conditions
Bethlem Myopathy
Sponsor
Ruth Salim
Enrollment
20
Locations
1
Primary Endpoint
Muscle fat fraction
Last Updated
7 years ago

Overview

Brief Summary

Collagen VI-related diseases include Bethlem myopathy and Ulrich dystrophy. They are both caused by decreased levels or a lack of collagen VI. The first symptoms can be present at birth as joint laxity and hypotonic muscles; often seen with luxation of the hip and scoliosis. During childhood, patients may develop contractures of fingers, wrists, elbows and ankles. Muscle weakness often appears in childhood to early adulthood and is progressive. It often results in walking difficulties.

There is no treatment available for Behtlem and Ulrich dystrophies.

The primary aim of this study is to investigate the pattern of involved muscles, the function and quality of the muscles and the disease severity using MRI.

Detailed Description

See above

Registry
clinicaltrials.gov
Start Date
September 1, 2018
End Date
May 1, 2019
Last Updated
7 years ago
Study Type
Interventional
Study Design
Single Group
Sex
All

Investigators

Sponsor
Ruth Salim
Responsible Party
Sponsor Investigator
Principal Investigator

Ruth Salim

Medical student

Rigshospitalet, Denmark

Eligibility Criteria

Inclusion Criteria

  • confirmed Bethlem myopathy or Ulrich disease

Exclusion Criteria

  • All contraindications for undergoing an MRI scan

Outcomes

Primary Outcomes

Muscle fat fraction

Time Frame: One MRI scan pr subject (exam lasts approximately 60 min)

The Dixon MRI will be used to quantify the fat fraction in skeletal muscle

Secondary Outcomes

  • Muscle strength(Exam lasts approximately 40-60 min)

Study Sites (1)

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