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Twice Weekly Steroids and Exercise as Therapy for DMD

Primary Purpose

Duchenne Muscular Dystrophy (DMD)

Status

Recruiting

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Prednisone

In-home Exercise Training

Prednisone plus exercise

About this trial

This is an interventional supportive care trial for Duchenne Muscular Dystrophy (DMD) focused on measuring steroid side effects, low dose steroid regimen, exercise training therapy

Eligibility Criteria

5 Years - 9 Years (Child)MaleDoes not accept healthy volunteers

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-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

Sites / Locations

University of FloridaRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

No Intervention

Active Comparator

Arm Label

Daily Glucocorticoid (GC)

Twice weekly glucocorticoid with or without exercise

Daily glucocorticoid with exercise

Arm Description

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.

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.

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Full Information

NCT ID

NCT04322357

First Posted

March 24, 2020

Last Updated

May 30, 2023

Sponsor

University of Florida

Collaborators

U.S. Army Medical Research and Development Command

1. Study Identification

Unique Protocol Identification Number

NCT04322357

Brief Title

Twice Weekly Steroids and Exercise as Therapy for DMD

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

May 2023

Overall Recruitment Status

Recruiting

Study Start Date

July 30, 2020 (Actual)

Primary Completion Date

October 7, 2023 (Anticipated)

Study Completion Date

October 7, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

University of Florida

Collaborators

U.S. Army Medical Research and Development Command

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Duchenne Muscular Dystrophy (DMD)

Keywords

steroid side effects, low dose steroid regimen, exercise training therapy

7. Study Design

Primary Purpose

Supportive Care

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

89 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Daily Glucocorticoid (GC)

Arm Type

No Intervention

Arm Description

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.

Arm Title

Twice weekly glucocorticoid with or without exercise

Arm Type

Active Comparator

Arm Description

Arm Title

Daily glucocorticoid with exercise

Arm Type

Active Comparator

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Prednisone

Other Intervention Name(s)

Glucocorticoid (GC)

Intervention Description

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

Intervention Type

Behavioral

Intervention Name(s)

In-home Exercise Training

Intervention Description

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

Drug

Intervention Name(s)

Prednisone plus exercise

Intervention Description

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

Primary Outcome Measure Information:

Title

Change in BMI

Description

Participant body mass index change (weight and height will be combined to report BMI in kg/m^2) over the course of one year

Time Frame

Baseline up to 12 months

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

5 Years

Maximum Age & Unit of Time

9 Years

Accepts Healthy Volunteers

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Tanja Taivassalo, PhD

Phone

(352) 294-8748

ttaivassalo@ufl.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Tanja Taivassalo, MD

Organizational Affiliation

University of Florida

Official's Role

Principal Investigator

Facility Information:

Facility Name

University of Florida

City

Gainesville

State/Province

Florida

ZIP/Postal Code

32610

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Tanja Taivassalo

Phone

352-294-8748

ttaivassalo@ufl.edu

First Name & Middle Initial & Last Name & Degree

Tanja Taivassalo

12. IPD Sharing Statement

Plan to Share IPD

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Twice Weekly Steroids and Exercise as Therapy for DMD

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