Twice Weekly Steroids and Exercise as Therapy for DMD

Duchenne Muscular Dystrophy (DMD) Clinical Trial

Official title:

Impact and Interplay of Corticosteroid Regimen and Exercise Training on DMD Muscle Function

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04322357
• Other study ID #: IRB201901339
• Secondary ID: MD 180023OCR2714
• Status: Recruiting
• Phase: Phase 2
• Start date: July 30, 2020
• Est. completion date: October 7, 2025

Study information

• Verified date: January 2024
• Source: University of Florida
• Contact: Tanja Taivassalo, PhD
• Phone: (352) 294-8748
• Email: ttaivassalo[@]ufl.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical 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). 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 89
• Est. completion date: October 7, 2025
• Est. primary completion date: October 7, 2024
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years to 9 Years

Eligibility

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy (DMD)
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

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

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.

Locations

Country	Name	City	State
United States	University of Florida	Gainesville	Florida

Sponsors (2)

Lead Sponsor	Collaborator
University of Florida	U.S. Army Medical Research and Development Command

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Baseline up to 12 months