United States, New Zealand, United Kingdom80 participantsStarted 2022-04-13
Plain-language summary
This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)
Who can participate
Age range
6 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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
. 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
Exclusion criteria
. 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)
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To assess the natural history of measures of muscle function in BMD
Timeframe: Through study completion, an average of 2 years
2
4-Stair Climb
Timeframe: Through study completion, an average of 2 years
3
100-Meter Timed Test
Timeframe: Through study completion, an average of 2 years
4
PERFORMANCE OF UPPER LIMB 2.0 (PUL)
Timeframe: Through study completion, an average of 2 years
5
HAND HELD DYNAMOMETRY (HHD) AND GRIP
Timeframe: Through study completion, an average of 2 years
6
TIMED UP-AND-GO (TUG)
Timeframe: Through study completion, an average of 2 years
7
Measures of Pulmonary Function (Seated and supine FVC)
Timeframe: Through study completion, an average of 2 years
. 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)
. Loss of ambulation prior to age 16
Measures of Pulmonary Function (MEP and MIP)
Timeframe: Through study completion, an average of 2 years
9
Measures of Pulmonary Function (other)
Timeframe: Through study completion, an average of 2 years
10
Measure of ejection fraction (ECHO)
Timeframe: Through study completion, an average of 2 years
11
Measure of systolic and diastolic function (ECHO)
Timeframe: Through study completion, an average of 2 years