Could a lower steroid dose plus exercise help boys with duchenne?
NCT ID NCT04322357
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looked at whether giving a steroid called prednisone twice a week at a lower dose, along with exercise training, could help boys with Duchenne muscular dystrophy (DMD) maintain muscle strength and function. The goal was to find a treatment that works as well as daily steroids but with fewer side effects. The study included 21 boys aged 5 to 9 with confirmed DMD.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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21 people
The number who actually took part.
- Started
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Jul 2020
- Finished
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Sep 2025
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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5 to 9 years
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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 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 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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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University of Florida
Gainesville, Florida, 32610, United States
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Other studies related to the condition(s) this trial covers.
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