welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
- log in
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: April 09, 2020
start date: April 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
Aerobic Exercise in Duchenne Muscular DystrophyDuchenne Muscular Dystrophy (DMD) is the...
CoQ10 and Prednisone in Non-Ambulatory DMDThis study will help determine if CoQ10 ...
Precision Medicine and Exercise Therapy in Duchenne Muscular DystrophyPrecision medicine is being discussed an...
Finding the Optimum Regimen for Duchenne Muscular DystrophyThe Finding the Optimum Regimen for Duch...
Moderate exercise improves function and increases adiponectin in the mdx mouse model of muscular dystrophyThe loss of dystrophin produces a mechan...
Regular Physical Exercise in Duchenne Muscular DystrophyThis study examine whether an evidence-b...
Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophyObjective: To assess safety and efficac...
Stretches for Duchenne Muscular Dystrophyhttps://www.youtube.com/watch?v=6eHLt3KA...
Voluntary Exercise Improves Muscle Function and Does not Exacerbate Muscle and Heart Pathology in Aged Duchenne Musc...Duchenne muscular dystrophy is a severe ...