Twice Weekly Steroids and Exercise as Therapy for DMD

Sponsor
University of Florida (Other)
Overall Status
Recruiting
CT.gov ID
NCT04322357
Collaborator
U.S. Army Medical Research and Development Command (U.S. Fed)
89
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Study Details

Study Description

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
  • Drug: Prednisone
  • Behavioral: In-home Exercise Training
  • Drug: Prednisone plus exercise
Phase 2

Study Design

Study Type:
Interventional
Anticipated 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 :
Jul 30, 2020
Anticipated Primary Completion Date :
Oct 7, 2023
Anticipated Study Completion Date :
Oct 7, 2023

Arms and Interventions

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.

    Outcome Measures

    Primary Outcome Measures

    1. Change in BMI [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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    5 Years to 8 Years
    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-na├»ve 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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of Florida Gainesville Florida United States 32610

    Sponsors and Collaborators

    • University of Florida
    • U.S. Army Medical Research and Development Command

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    University of Florida
    ClinicalTrials.gov Identifier:
    NCT04322357
    Other Study ID Numbers:
    • IRB201901339
    • MD 180023
    • OCR27142
    First Posted:
    Mar 26, 2020
    Last Update Posted:
    Mar 16, 2022
    Last Verified:
    Feb 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by University of Florida
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Mar 16, 2022