Weekend Steroids and Exercise as Therapy for DMD

study id #: NCT04322357

condition: Duchenne Muscular Dystrophy (DMD)

status: not yet recruiting

purpose:

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

results: https://clinicaltrials.gov/ct2/show/results/NCT04322357

last updated: September 22, 2020

start date: October 2020

estimated completion: October 2022

phase of development: Phase 2

size / enrollment: 65

primary outcomes:

• 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

inclusion criteria:

• Diagnosis of DMD confirmed by 1) clinical history with features before the age of five, 2) physical examination, 3) elevated serum creatine kinase level and 4) absence of dystrophin expression, as determined by immunostain or Western blot (<2%) and/or DNA confirmation of dystrophin mutation.
• 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

exclusion criteria:
• 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

sponsor: University of Florida

contacts: Tanja Taivassalo, 352-294-8748, ttaivassalo@ufl.edu

investigators: Tanja Taivassalo, 352-294-8748, ttaivassalo@ufl.edu