Defining Endpoints in Becker Muscular Dystrophy
This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)
Conditions:
🦠 Becker Muscular Dystrophy 🦠 Muscular Dystrophies 🦠 Muscular Dystrophy in Children 🦠 Muscular Dystrophy, Becker
🗓️ Study Start (Actual) 13 April 2022
🗓️ Primary Completion (Estimated) May 2025
✅ Study Completion (Estimated) May 2026
👥 Enrollment (Estimated) 80
🔬 Study Type OBSERVATIONAL
📊 Phase N/A
Locations:
📍 Orange, California, United States
📍 Aurora, Colorado, United States
📍 Iowa City, Iowa, United States
📍 Kansas City, Kansas, United States
📍 Baltimore, Maryland, United States
📍 Minneapolis, Minnesota, United States
📍 Saint Louis, Missouri, United States
📍 Columbus, Ohio, United States
📍 Richmond, Virginia, United States
📍 Auckland, New Zealand
📍 Newcastle Upon Tyne, United Kingdom

📋 Eligibility Criteria

Description

    Inclusion Criteria:

    • For ages 6-12
    • 1. Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD)
    • 2. Genetic confirmation of an in-frame dystrophin mutation
    • 3. Ambulatory
    • 4. Willing and able to give informed consent and follow all procedures and requirements
    • For ages 13 and older
    • 1. Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD)
    • 2. Genetic confirmation of a dystrophin mutation
    • 3. 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 Criteria:

    • For ages 6-12
    • 1. Out of frame dystrophin mutation
    • 2. Use of chronic corticosteroids at baseline, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population
    • 3. Non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics)
    • 4. \>16 hours of ventilatory support
    • 5. 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.
    • 6. Under the age of 6 at time of enrollment
    • 7. 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
    • 1. Loss of ambulation prior to age 16
    • 2. Use of chronic corticosteroids, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population
    • 3. Less than 30% of the overall population will be non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics)
    • 4. \>16 hours of ventilatory support
    • 5. Subjects aged 13-16 only: time to rise \>10 seconds
    • 6. For MR Cohort: Have contraindications to MRI or MRS (e.g., non-MR compatible implanted medical devices or severe claustrophobia)
Ages Eligible for Study: 6 Years to N/A (CHILD, ADULT, OLDER_ADULT)
Sexes Eligible for Study: MALE
Accepts Healthy Volunteers: No

🗓️ Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

  • First Submitted 25 January 2022
  • First Submitted that Met QC Criteria 15 February 2022
  • First Posted 25 February 2022

Study Record Updates

  • Last Update Submitted that Met QC Criteria 1 July 2024
  • Last Update Posted 3 July 2024
  • Last Verified July 2024