Twice Weekly Steroids and Exercise as Therapy for DMD

NCT04322357 | Completed Phase 2 Results DMC FDA Drug

• 3 other identifiers: IRB201901339MD 180023OCR27142
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

novel steroid regimen; twice-weekly prednisone; exercise training; leg cycling; isometric strengthening

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (3)

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.

Drug: Prednisone

Daily glucocorticoid with exercise

ACTIVE COMPARATOR

Patients on daily glucocorticoids will undergo 6 months of structured, supervised and home-based exercise training.

Drug: Prednisone; Behavioral: Exercise Training on standard steroid regimen; Drug: Exercise training on twice-weekly steroids

Interventions

Prednisone DRUG

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

Also known as: Glucocorticoid (GC)

Daily glucocorticoid with exercise; Twice weekly glucocorticoid with or without exercise

Exercise training on twice-weekly steroids DRUG

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

Also known as: no other names apply

Daily glucocorticoid with exercise

Exercise Training on standard steroid regimen BEHAVIORAL

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.

Also known as: no other names apply

Daily glucocorticoid with exercise

Eligibility Criteria

• Age: 5 Years - 9 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

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

Study Sites (1)

University of Florida

Gainesville, Florida, 32610, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Prednisone; Glucocorticoids

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Physiological Effects of Drugs; Pharmacologic Actions; Chemical Actions and Uses

Results Point of Contact

• Title: Research Associate Professor
• Organization: University of Florida

ttaivassalo@ufl.edu

352-339-6666

Study Officials

• Tanja Taivassalo, MD

  University of Florida

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 24, 2020
• First Posted: March 26, 2020
• Study Start: July 30, 2020
• Primary Completion: December 4, 2024
• Study Completion: September 4, 2025
• Last Updated: March 13, 2026
• Results First Posted: March 13, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)