Status:
ACTIVE_NOT_RECRUITING
Twice Weekly Steroids and Exercise as Therapy for DMD
Lead Sponsor:
University of Florida
Collaborating Sponsors:
U.S. Army Medical Research and Development Command
Conditions:
Duchenne Muscular Dystrophy (DMD)
Eligibility:
MALE
5-9 years
Phase:
PHASE2
Brief Summary
The study team will determine the potential of low dose twice weekly prednisone and whether exercise training can synergize to delay disease progression and improve muscle strength/physical function i...
Eligibility Criteria
Inclusion
- 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 9 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 9 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
- 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
Key Trial Info
Start Date :
July 30 2020
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 7 2025
Estimated Enrollment :
89 Patients enrolled
Trial Details
Trial ID
NCT04322357
Start Date
July 30 2020
End Date
October 7 2025
Last Update
February 27 2025
Active Locations (1)
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1
University of Florida
Gainesville, Florida, United States, 32610