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Weekend Steroids and Exercise as Therapy for DMD
study id #: NCT04322357
condition: Duchenne Muscular Dystrophy (DMD)
status: not yet recruitingpurpose:
The study team will determine the potential of low dose weekend prednisone and whether exercise training can synergize to delay disease progression and improve muscle strength/physical function in boys with Duchenne muscular dystrophy (DMD). Current standard of care (daily prednisone) is associated with adverse side effects. Evidence from DMD mouse models suggest that weekend dosing provides same efficacy without side effects. Appropriate exercise may also benefit but this area has not been adequately explored.
intervention: Drug: Prednisone, Behavioral: In-home Exercise Training
mechanism of action: Glucocorticoid to delay decline in muscle strength
last updated: September 22, 2020
start date: October 2020
estimated completion: October 2022
phase of development: Phase 2
size / enrollment: 65
- Change in BMI [ Time Frame: Baseline up to 12 months ]
Participant body mass index change (weight and height will be combined to report BMI in kg/m^2) over the course of one year
• Eligible Sexes: male
• Age 5.0 to 8 years: a lower age limit of 5.0 years is selected as children younger than that are likely unable to cooperate and comply with all of the exercise measures as needed. An upper age limit of 8 years has been set as boys with DMD tend to reach a rapid progression into a late ambulatory phase soon after this age.
• Ambulatory at the time of the first visit, defined as the ability to walk for at least 100 m without an external assistive device and able to climb four stairs.
• Aim 1 only: GC-naïve at baseline (and prior 6 months)
• Aim 2 only: on stable daily GC regimen for 6 months prior to baseline
• Contraindication to an MR examination (e.g. aneurysm clip, severe claustrophobia, magnetic implants)
• Presence of unstable medical problems, significant concomitant illness including cardiomyopathy or cardiac conduction abnormalities
• Presence of a secondary condition that impacts muscle function or muscle metabolism (e.g. myasthenia gravis, endocrine disorder, mitochondrial disease)
• Presence of a secondary condition leading to developmental delay or impaired motor control (e.g. cerebral palsy)
• Presence of an unstable medical condition (e.g. uncontrolled seizure disorder)
• Behavioral problems causing an inability to cooperate during testing or understand exercise instruction
• Participation in other forms of drug or gene therapy during the period of the study
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