Status:
ACTIVE_NOT_RECRUITING
Defining Endpoints in Becker Muscular Dystrophy
Lead Sponsor:
Virginia Commonwealth University
Collaborating Sponsors:
Edgewise Therapeutics, Inc.
Conditions:
Becker Muscular Dystrophy
Muscular Dystrophies
Eligibility:
MALE
6+ years
Brief Summary
This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)
Detailed Description
Becker Muscular Dystrophy (BMD) is most frequently due to in-frame mutations in the dystrophin gene that are associated with reduced levels of frequently shortened dystrophin, though other mutations m...
Eligibility Criteria
Inclusion
- For ages 6-12
- Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD)
- Genetic confirmation of an in-frame dystrophin mutation
- Ambulatory
- Willing and able to give informed consent and follow all procedures and requirements
- For ages 13 and older
- Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD)
- Genetic confirmation of a dystrophin mutation
- Willing and able to give informed consent and follow all procedures and requirements
- For participants in the MRI substudy:
- 1\. Ambulatory, defined as able to walk 10 meters without assistive devices (orthotics allowed)
Exclusion
- For ages 6-12
- Out of frame dystrophin mutation
- Use of chronic corticosteroids at baseline, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population
- Non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics)
- \>16 hours of ventilatory support
- Any other illness that would interfere with the ability to undergo safe testing or would interfere with interpretation of the results in the opinion of the site investigator.
- Under the age of 6 at time of enrollment
- For MR Cohort: Have contraindications to MRI or MRS (e.g., non-MR compatible implanted medical devices or severe claustrophobia)
- For ages 13 and older
- Loss of ambulation prior to age 16
- Use of chronic corticosteroids, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population
- Less than 30% of the overall population will be non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics)
- \>16 hours of ventilatory support
- Subjects aged 13-16 only: time to rise \>10 seconds
- For MR Cohort: Have contraindications to MRI or MRS (e.g., non-MR compatible implanted medical devices or severe claustrophobia)
Key Trial Info
Start Date :
April 13 2022
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
May 1 2026
Estimated Enrollment :
80 Patients enrolled
Trial Details
Trial ID
NCT05257473
Start Date
April 13 2022
End Date
May 1 2026
Last Update
June 8 2025
Active Locations (11)
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1
University of California, Irvine
Orange, California, United States, 92868
2
University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States, 80045
3
University of Iowa
Iowa City, Iowa, United States, 52242
4
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160