NCT04322357CompletedPhase 2
Twice Weekly Steroids and Exercise as Therapy for DMD
Impact and Interplay of Corticosteroid Regimen and Exercise Training on DMD Muscle Function
Source: ClinicalTrials.gov · Last updated 13 Mar 2026
Brief summary (registry, in English)
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 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 weekly dosing provides same efficacy without side effects. Appropriate exercise may also benefit but this area has not been adequately explored.
- Status (registry)
- Completed
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 21 (actual)
- First submitted
- 24 Mar 2020
- Start date
- 30 Jul 2020
- Primary completion date
- 4 Dec 2024
- Lead sponsor
- University of Florida
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT04322357
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 21
- From 30 Jul 2020 to the primary completion date, 4 Dec 2024
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
- Participation in other forms of drug or gene therapy during the period of the study
Other illness
- 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)
Other
- Behavioral problems causing an inability to cooperate during testing or understand exercise instruction
Who the study is looking for
- 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
Age in the registry: 5 years to 9 years · Sex: Male · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of Florida Coordinator of this site? Confirm its status | Gainesville | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.