Twice Weekly Steroids and Exercise as Therapy for DMD

January 30, 2024 updated by: University of Florida

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

89

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Florida
      • Gainesville, Florida, United States, 32610
        • Recruiting
        • University of Florida
        • Contact:
        • Principal Investigator:
          • Tanja Taivassalo

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years to 8 years (Child)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Supportive Care
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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 Names:
  • 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 Names:
  • 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Baseline up to 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Florida

Collaborators

U.S. Army Medical Research and Development Command

Investigators

  • Principal Investigator: Tanja Taivassalo, MD, University of Florida

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 30, 2020

Primary Completion (Estimated)

October 7, 2024

Study Completion (Estimated)

October 7, 2025

Study Registration Dates

First Submitted

March 24, 2020

First Submitted That Met QC Criteria

March 24, 2020

First Posted (Actual)

March 26, 2020

Study Record Updates

Last Update Posted (Actual)

January 31, 2024

Last Update Submitted That Met QC Criteria

January 30, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IRB201901339
  • MD 180023 (Other Grant/Funding Number: U.S. Army Medical Research and Development Command)
  • OCR27142 (Other Identifier: UF OnCore)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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