Duchenne Muscular Dystrophy (DMD)Phase 2NCT04322357
Twice Weekly Steroids and Exercise as Therapy for DMD
A Phase 2 study testing Prednisone, Exercise Training on standard steroid regimen and Exercise training on twice-weekly steroids in people with Duchenne Muscular Dystrophy (DMD), run by University of Florida at 1 site in United States.
Trial 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 in boys with Duchenne muscular dystrophy (DMD). Read more about this study
- Who is it for?
- Ages 5 to 9 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- Prednisone, Exercise Training on standard steroid regimen and Exercise training on twice-weekly steroids
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Jul 2020to 4 Sep 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 9 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Impact and Interplay of Corticosteroid Regimen and Exercise Training on DMD Muscle Function
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.
Read the detailed description
This innovative proposal focuses on developing an efficacious therapeutic strategy involving low dose twice weekly glucocorticoid (GC) administration and exercise training for boys affected with Duchenne muscular dystrophy (DMD), a currently incurable disease characterized by rapidly progressive muscle weakness, early loss of ambulation and death. While GC are the only proven treatment to reduce fibrosis and delay loss of ambulation in DMD, chronic daily administration (which is most commonly prescribed) is associated with adverse, often debilitating effects. As an alternate dosing regimen, weekend-only use was shown to retain the benefits and have less impact on weight gain and linear growth, however high doses were used and associated with behavioral issues. Recent work in mice suggests that the same daily dose administered transiently may be effective and have a greater impact on gains in muscle mass, strength and resistance to fatigue compared to daily dosing due to differential effects on gene expression signaling pathways important for muscle remodeling. Exercise, which also induces signaling pathways that lead to remodeling in healthy muscle, may beneficially impact pathophysiology of DMD by recruiting compensatory pathways. Although high intensity or eccentric actions are damaging to dystrophic muscle, a few studies suggest that submaximal exercise is safe and may delay the loss of muscle function in boys with DMD. Despite these exploratory studies suggesting potential, there is a paucity of research on exercise, which reflects our current lack of understanding of specific exercise prescription parameters (type, intensity, target muscle groups) that may be safe and effective for patients with DMD, as well as lack of accessibility to exercise equipment that appropriately and sufficiently induces adaptation in dystrophic muscle. The objective of this work is to define an efficacious GC regimen with minimal side effects, and understand if exercise training can potentially delay disease progression, reverse secondary effects of disuse, and induce beneficial adaptations in boys with DMD.
AIM 1: To determine the 12-month impact of a low dose (0.75 mg/kg x 2 days of prednisone) regimen on weight gain, DMD muscle pathophysiology and physical function. We hypothesize that compared to the standard daily regimen, a twice-weekly regimen will have less impact on body mass index, and equal improvements in the 1-year change in physical function and muscle fat fraction.
AIM 2: To determine impact of a 6-month in-home, moderate intensity, leg exercise training program on muscle pathophysiology and physical function in DMD.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- Prednisone, Exercise Training on standard steroid regimen, Exercise training on twice-weekly steroids
- Keywords
- novel steroid regimen, twice-weekly prednisone, exercise training, leg cycling, isometric strengthening
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PrednisoneDrug
Also called Glucocorticoid (GC)
A 12-month treatment period with twice weekly, low-dose prednisone (dose of 0.75 mg/kg per day).
Exercise Training on standard steroid regimenBehavioural
Also called no other names apply
For boys on current standard of care (daily glucocorticoid use), 6-months in-home, remotely supervised exercise training program involving a combination of aerobic and isometric leg strength exercises.
Exercise training on twice-weekly steroidsDrug
Also called no other names apply
Twice weekly prednisone for 6 months followed by twice weekly prednisone plus exercise for 6 months.
| Study group | Role | What they receive |
|---|---|---|
| Daily Glucocorticoid (GC) | No intervention | Existing data from age-matched, ambulatory, on daily GC therapy, and similar exclusion criteria will be selected from the ImagingDMD database to serve as a historical control. |
| Twice weekly glucocorticoid with or without exercise | Active comparator | Patients will be randomized to one of 2 groups:
* Twice weekly prednisone alone for 12 months
* Twice weekly prednisone for 6 months followed by twice weekly prednisone plus 6 months of structured, supervised and home-based exercise training. Receives: Drug: Prednisone |
| Daily glucocorticoid with exercise | Active comparator | Patients on daily glucocorticoids will undergo 6 months of structured, supervised and home-based exercise training. Receives: Drug: Prednisone; Behavioral: Exercise Training on standard steroid regimen; Drug: Exercise training on twice-weekly steroids |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in BMI
Participant body mass index change (weight and height will be combined to report BMI in kg/m^2) over the course of one year
Measured: Baseline up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Mar 2020
- Study start30 Jul 2020
- Primary completion4 Dec 2024
- Study completion4 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- U.S. Army Medical Research and Development Command
- Responsible party
- Sponsor
- Investigators
- Tanja Taivassalo, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04322357
- Sponsor's protocol ID
- IRB201901339
- Other IDs
- MD 180023, OCR27142
- Status last verified
- Dec 2025
- First submitted
- 24 Mar 2020
- Last updated
- 13 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Impact and Interplay of Corticosteroid Regimen and Exercise Training on DMD Muscle Function
- ClinicalTrials.gov ID
- NCT04322357
- Acronym / sponsor ID
- IRB201901339
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- novel steroid regimen, twice-weekly prednisone, exercise training, leg cycling, isometric strengthening
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- U.S. Army Medical Research and Development Command
- Study start
- 30 Jul 2020 (actual)
- Primary completion
- 4 Dec 2024 (actual)
- Study completion
- 4 Sep 2025 (actual)
- First posted
- 26 Mar 2020 (actual)
- Results
- Posted 13 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This innovative proposal focuses on developing an efficacious therapeutic strategy involving low dose twice weekly glucocorticoid (GC) administration and exercise training for boys affected with Duchenne muscular dystrophy (DMD), a currently incurable disease characterized by rapidly progressive muscle weakness, early loss of ambulation and death. While GC are the only proven treatment to reduce fibrosis and delay loss of ambulation in DMD, chronic daily administration (which is most commonly prescribed) is associated with adverse, often debilitating effects. As an alternate dosing regimen, weekend-only use was shown to retain the benefits and have less impact on weight gain and linear growth, however high doses were used and associated with behavioral issues. Recent work in mice suggests that the same daily dose administered transiently may be effective and have a greater impact on gains in muscle mass, strength and resistance to fatigue compared to daily dosing due to differential effects on gene expression signaling pathways important for muscle remodeling. Exercise, which also induces signaling pathways that lead to remodeling in healthy muscle, may beneficially impact pathophysiology of DMD by recruiting compensatory pathways. Although high intensity or eccentric actions are damaging to dystrophic muscle, a few studies suggest that submaximal exercise is safe and may delay the loss of muscle function in boys with DMD. Despite these exploratory studies suggesting potential, there is a paucity of research on exercise, which reflects our current lack of understanding of specific exercise prescription parameters (type, intensity, target muscle groups) that may be safe and effective for patients with DMD, as well as lack of accessibility to exercise equipment that appropriately and sufficiently induces adaptation in dystrophic muscle. The objective of this work is to define an efficacious GC regimen with minimal side effects, and understand if exercise training can potentially delay disease progression, reverse secondary effects of disuse, and induce beneficial adaptations in boys with DMD.
AIM 1: To determine the 12-month impact of a low dose (0.75 mg/kg x 2 days of prednisone) regimen on weight gain, DMD muscle pathophysiology and physical function. We hypothesize that compared to the standard daily regimen, a twice-weekly regimen will have less impact on body mass index, and equal improvements in the 1-year change in physical function and muscle fat fraction.
AIM 2: To determine impact of a 6-month in-home, moderate intensity, leg exercise training program on muscle pathophysiology and physical function in DMD.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Daily Glucocorticoid (GC) | No intervention | –Existing data from age-matched, ambulatory, on daily GC therapy, and similar exclusion criteria will be selected from the ImagingDMD database to serve as a historical control. |
| Twice weekly glucocorticoid with or without exercise | Active comparator | Drug: PrednisonePatients will be randomized to one of 2 groups: * Twice weekly prednisone alone for 12 months * Twice weekly prednisone for 6 months followed by twice weekly prednisone plus 6 months of structured, supervised and home-based exercise training. |
| Daily glucocorticoid with exercise | Active comparator | Drug: Prednisone, Behavioral: Exercise Training on standard steroid regimen, Drug: Exercise training on twice-weekly steroidsPatients on daily glucocorticoids will undergo 6 months of structured, supervised and home-based exercise training. |
Who may qualify
- Ages
- 5 to 9 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (7 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PrednisoneAlso: Glucocorticoid (GC)A 12-month treatment period with twice weekly, low-dose prednisone (dose of 0.75 mg/kg per day). | Drug | Daily glucocorticoid with exercise, Twice weekly glucocorticoid with or without exercise |
| Exercise Training on standard steroid regimenAlso: no other names applyFor boys on current standard of care (daily glucocorticoid use), 6-months in-home, remotely supervised exercise training program involving a combination of aerobic and isometric leg strength exercises. | Behavioural | Daily glucocorticoid with exercise |
| Exercise training on twice-weekly steroidsAlso: no other names applyTwice weekly prednisone for 6 months followed by twice weekly prednisone plus exercise for 6 months. | Drug | Daily glucocorticoid with exercise |
Primary outcome
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Tanja Taivassalo, MD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Prednisone, Exercise Training on standard steroid regimen and Exercise training on twice-weekly steroids in people with Duchenne Muscular Dystrophy (DMD), run by University of Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in BMI (time frame: Baseline up to 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Mar 2026.