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Twice Weekly Steroids and Exercise as Therapy for DMD

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ClinicalTrials.gov Identifier: NCT04322357
Recruitment Status : Recruiting
First Posted : March 26, 2020
Last Update Posted : March 16, 2022
U.S. Army Medical Research and Development Command
Information provided by (Responsible Party):
University of Florida

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 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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy (DMD) Drug: Prednisone Behavioral: In-home Exercise Training Drug: Prednisone plus exercise Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 89 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official Title: Impact and Interplay of Corticosteroid Regimen and Exercise Training on DMD Muscle Function
Actual Study Start Date : July 30, 2020
Estimated Primary Completion Date : October 7, 2023
Estimated Study Completion Date : October 7, 2023

Arm Intervention/treatment
No Intervention: Daily Glucocorticoid (GC)
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.
Active Comparator: Twice weekly glucocorticoid with or without exercise

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.
Drug: Prednisone
A 12-month treatment period with twice weekly, low-dose prednisone (dose of 0.75 mg/kg per day).
Other Name: Glucocorticoid (GC)

Active Comparator: Daily glucocorticoid with exercise
Patients on daily glucocorticoids will undergo 6 months of structured, supervised and home-based exercise training.
Drug: Prednisone
A 12-month treatment period with twice weekly, low-dose prednisone (dose of 0.75 mg/kg per day).
Other Name: Glucocorticoid (GC)

Behavioral: In-home Exercise Training
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.

Drug: Prednisone plus exercise
Twice weekly prednisone for 6 months followed by twice weekly prednisone plus exercise for 6 months.

Primary Outcome Measures :
  1. 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

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 8 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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 8 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 8 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04322357

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Contact: Tanja Taivassalo, PhD (352) 294-8748 ttaivassalo@ufl.edu

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United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Contact: Tanja Taivassalo    352-294-8748    ttaivassalo@ufl.edu   
Principal Investigator: Tanja Taivassalo         
Sponsors and Collaborators
University of Florida
U.S. Army Medical Research and Development Command
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Principal Investigator: Tanja Taivassalo, MD University of Florida
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Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT04322357    
Other Study ID Numbers: IRB201901339
MD 180023 ( Other Grant/Funding Number: U.S. Army Medical Research and Development Command )
OCR27142 ( Other Identifier: UF OnCore )
First Posted: March 26, 2020    Key Record Dates
Last Update Posted: March 16, 2022
Last Verified: February 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by University of Florida:
steroid side effects, low dose steroid regimen, exercise training therapy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Anti-Inflammatory Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents