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Could a lower steroid dose plus exercise help boys with duchenne?

NCT ID NCT04322357

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

21 people

The number who actually took part.

Started

Jul 2020

Finished

Sep 2025

Lead sponsor

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

5 to 9 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

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

  • University of Florida

    Gainesville, Florida, 32610, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.