Becker Muscular Dystrophy: analysis of diversity in disease severity

Recruiting

• Conditions: Becker; muscular dystrophy; 10029317

• Registration Number: NL-OMON35006
• Lead Sponsor: eids Universitair Medisch Centrum

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 6 May 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 60

Inclusion Criteria

1. diagnosis of Muscular Dystrophy, defined by:
- male gender AND
- progressive muscular weakness AND
- elevated serum CPK-levels AND
- dystrophic characteristics on muscle biopsy OR
- reduced amount of dystrophin in muscle biopsy OR
- in frame mutation in DMD gene
2. Age over 18 years

Exclusion Criteria

Exlusion for MRI
- claustrophobia
- pacemaker and defibrillators
- nerve stimulators
- intracranial clips
- intraorbital or intraocular metallic fragments
- cochlear implants
- ferromagnetic implants (eg. thoracic implant for scoliosis)
- inability to lie supine during less than 60 minutes

Study & Design

• Study Type: Observational invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>1. Disease course, based on data collected from a structured medical history<br /><br>and information from other treating physicians.<br /><br><br /><br>2. Biomarkers from blood<br /><br><br /><br>3. Strength and Functional Assessment<br /><br><br /><br>4. Muscle MRI<br /><br><br /><br>5. Muscle biopsy parameters<br /><br><br /><br>6. Echocardiography</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>NA</p><br>