Twice Weekly Steroids and Exercise as Therapy for DMD

NCT ID: NCT04322357

Last Updated: 2025-02-27

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 89 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-07-30
• Study Completion Date: 2025-10-07

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)

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: NONE

Study Groups

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.

Group Type: NO_INTERVENTION

No interventions assigned to this group

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.

Group Type: ACTIVE_COMPARATOR

Prednisone

Intervention Type: DRUG

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

Daily glucocorticoid with exercise

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

Group Type: ACTIVE_COMPARATOR

Prednisone

Intervention Type: DRUG

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

In-home Exercise Training

Intervention Type: 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.

Prednisone plus exercise

Intervention Type: DRUG

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

Interventions

Prednisone

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

Intervention Type: DRUG

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.

Intervention Type: BEHAVIORAL

Prednisone plus exercise

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

Intervention Type: DRUG

Other Intervention Names

Glucocorticoid (GC)

Eligibility Criteria

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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 9 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

U.S. Army Medical Research and Development Command

FED

Sponsor Role: collaborator

University of Florida

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Tanja Taivassalo, MD

Role: PRINCIPAL_INVESTIGATOR

University of Florida

Locations

University of Florida

Gainesville, Florida, United States

Countries

United States

Other Identifiers

MD 180023

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

OCR27142

Identifier Type: OTHER

Identifier Source: secondary_id

IRB201901339

Identifier Type: -

Identifier Source: org_study_id