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An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-

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ClinicalTrials.gov Identifier: NCT02708784
Recruitment Status : Unknown
Verified November 2015 by University of Aarhus.
Recruitment status was:  Enrolling by invitation
First Posted : March 15, 2016
Last Update Posted : March 15, 2016
Sponsor:
Information provided by (Responsible Party):
University of Aarhus

Brief Summary:

The aim of the project is to develop new Magnetic Resonance (MR) imaging techniques for better diagnosis and monitoring of patients with muscular disorders.

Muscle quality in patients with Late Onset Pompe Disease (Acid Maltase Deficiency type 2) and in patients with Myotonica Dystrophy will be evaluated, by determining muscle strength in relation to muscle size and muscle strength in relations to fat-muscle ratio.


Condition or disease
Glycogen Storage Disease Type 2 Dystrophia Myotonica

  Show Detailed Description

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Study Type : Observational
Estimated Enrollment : 60 participants
Time Perspective: Cross-Sectional
Official Title: Function, Structure and Quality of Striated Muscles in Patients With Muscular Diseases - an MRI Study on Pompe Disease and Dystrophia Myotonica
Study Start Date : September 2015
Estimated Primary Completion Date : August 2016
Estimated Study Completion Date : September 2016


Group/Cohort
Pompe disease
Patients will perform muscle strength measurement with dynamometer and MR-imaging. After 8 months the investigations will be repeated.
Myotonic Dystrophy
Patients will perform muscle strength measurement with dynamometer and MR-imaging. The investigations will not be repeated after 8 months.
Healthy controls
Patients will perform muscle strength measurement with dynamometer and MR-imaging. The investigations will not be repeated after 8 months.



Primary Outcome Measures :
  1. Muscle strength in Newton*meter [ Time Frame: one year ]
    Muscle strength in measured by dynamometry, and the used outcome is the "peak torque".

  2. Muscle to fat ratio [ Time Frame: one year ]
    Calculated from the MR-images, is a measure for muscle quality. The calculations are based on the signal intensity of the fat and water Dixon MR-images.

  3. Volume ratio [ Time Frame: one year ]
    Defined from the MR-images, is a measure for muscle quality.


Secondary Outcome Measures :
  1. 6 minutes walking test [ Time Frame: one year ]
    Only for Pompe disease participants

  2. Forced Vital Function [ Time Frame: on year ]
    Only for Pompe disease participants



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population

Patients will be recruited trough their attendance to the Hospital. Pompe disease patients will be recruited from all the treating hospitals in Denmark and from the University hospital of Münster. Patients with Myotonic Dystrophy will be recruited trough their attendance at Aarhus University Hospital.

Healthy controls will be recruited from the general Danish population.

Criteria

Inclusion Criteria:

  • Diagnosed Pompe disease patients, whether they are in treatment with ERT or not, or Diagnosed Dystrophia Myotonica patients.
  • Healthy controls, have to be sex and age-matched to patients with muscular disease.

Exclusion Criteria:

  • Age under 18 years.
  • The subjects must be able to perform an MR-scanning. Moreover they have to sign and respect the MR security rules "Kontrol skema før MR undersøgelse" and "Patient information i forbindelse med MR scanning".
  • The subjects must be capable of performing the muscle strength test by the Dynamometer.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02708784


Locations
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Denmark
Department of Neurology, Aarhus University Hospital
Aarhus C, Denmark, 8000
MR centre, Skejby University Hospital.
Aarhus N, Denmark, 8200
Sponsors and Collaborators
University of Aarhus
Investigators
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Study Chair: Henning Andersen, Professor Aarhus University Hospital

Publications:

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Responsible Party: University of Aarhus
ClinicalTrials.gov Identifier: NCT02708784     History of Changes
Other Study ID Numbers: PsDM15RMBH
First Posted: March 15, 2016    Key Record Dates
Last Update Posted: March 15, 2016
Last Verified: November 2015
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by University of Aarhus:
Glykogen storage disease type 2)
Dystrophia Myotonica
Muscular disorders
MR-imaging
Muscle strength
Myopathies

Additional relevant MeSH terms:
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Myotonic Dystrophy
Muscular Dystrophies
Myotonic Disorders
Glycogen Storage Disease Type II
Glycogen Storage Disease
Muscular Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Muscular Disorders, Atrophic
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases