What side effects are associated with this type of intervention?

Common treatments for Duchenne Muscular Dystrophy (DMD) include glucocorticoids like prednisone and exercise training. Prednisone, used for its anti-inflammatory and immunosuppressive effects, can cause side effects such as weight gain, osteoporosis, hypertension, glucose intolerance, and increased risk of infections. Exercise training enhances muscle strength and physical function but must be carefully managed to avoid overexertion and potential muscle damage. Combining low-dose, twice-weekly prednisone with exercise training aims to reduce these side effects while maintaining therapeutic benefits.

What prior FDA approvals exist?

The FDA has approved several treatments for Duchenne Muscular Dystrophy (DMD) that focus on anti-inflammatory and immunosuppressive effects, as well as enhancing muscle strength and physical function. Prednisone, a corticosteroid, is commonly used to slow muscle degeneration and improve strength. Eteplirsen, an exon-skipping drug, has been approved to increase dystrophin production in patients with specific genetic mutations. Additionally, deflazacort, another corticosteroid, has been approved for its benefits in improving muscle strength and function. These treatments aim to manage symptoms and improve quality of life for individuals with DMD.

What other types of research exist?

Promising novel alternatives for DMD treatment in 2024 include: 1) Eteplirsen, an exon-skipping therapy that increases dystrophin production and has shown benefits in the six-minute walk test. 2) Gene therapy approaches, such as micro-dystrophin gene transfer, which aim to restore dystrophin expression. 3) CRISPR/Cas9 gene editing, which holds potential for correcting genetic mutations directly. These therapies are in various stages of clinical trials and have shown encouraging results in improving muscle function and slowing disease progression.

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Corticosteroid

Twice weekly glucocorticoid with or without exercise for Duchenne Muscular Dystrophy

Phase 2

Recruiting

Led By Tanja Taivassalo, MD

Research Sponsored by University of Florida

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of DMD confirmed by clinical history, physical examination, elevated serum creatine kinase level, absence of dystrophin expression, and/or DNA confirmation of dystrophin mutation

Age 5.0 to 9 years

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline up to 12 months

Awards & highlights

Study Summary

This trial will study if low doses of prednisone given less often and exercise training can help boys with Duchenne muscular dystrophy.

Who is the study for?

Boys aged 5 to 9 with confirmed Duchenne muscular dystrophy (DMD) who can walk at least 100 meters and climb stairs without help. They must not have been on steroids for the past six months or be on a stable steroid regimen if participating in different parts of the trial. Those with unstable medical conditions, severe heart issues, behavioral problems that affect testing cooperation, or other muscle-impacting diseases cannot join.Check my eligibility

What is being tested?

The study is testing low dose prednisone given twice weekly combined with exercise training against the standard daily prednisone treatment for DMD. The goal is to see if this new approach slows disease progression and improves muscle strength without causing significant side effects.See study design

What are the potential side effects?

While daily prednisone use is known to cause adverse effects like weight gain, bone thinning, and high blood pressure, this trial aims to determine if a lower frequency dosage reduces these risks. Exercise may pose fatigue or strain but is considered beneficial when properly monitored.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with DMD based on tests and physical exams.

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I am between 5 and 9 years old.

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I was able to walk on my own during my first visit.

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I have not received any gastric cancer treatment before.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline up to 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline up to 12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to 12 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change in BMI

Trial Design

3Treatment groups

Active Control

Group I: Daily Glucocorticoid (GC)Active Control1 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.

Group II: Twice weekly glucocorticoid with or without exerciseActive Control1 Intervention

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 III: Daily glucocorticoid with exerciseActive Control3 Interventions

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

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Prednisone, a glucocorticoid, reduces inflammation and modulates the immune response, which helps slow muscle degeneration and improve muscle strength in Duchenne Muscular Dystrophy (DMD) patients. Exercise training enhances muscle strength and physical function by promoting muscle regeneration and reducing muscle wasting. These treatments are essential for DMD patients as they help maintain mobility and improve overall quality of life.

Dental pulp stem cells can improve muscle dysfunction in animal models of Duchenne muscular dystrophy.Treatment with human immunoglobulin G improves the early disease course in a mouse model of Duchenne muscular dystrophy.The proton pump inhibitor lansoprazole improves the skeletal phenotype in dystrophin deficient mdx mice.