Steroids + Exercise for Duchenne Muscular Dystrophy
Trial Summary
Do I have to stop taking my current medications for this trial?
The trial protocol does not specify if you need to stop taking your current medications. However, if you are participating in Aim 1, you must not have taken glucocorticoids (GC) for 6 months prior. If you are in Aim 2, you need to be on a stable daily GC regimen for 6 months before starting the trial.
Do I need to stop my current medications for the trial?
The trial does not specify if you need to stop your current medications. However, if you are part of Aim 1, you should not have taken glucocorticoids (a type of steroid) for at least 6 months before starting the trial. If you are part of Aim 2, you should have been on a stable daily glucocorticoid regimen for 6 months before the trial.
What data supports the idea that Steroids + Exercise for Duchenne Muscular Dystrophy is an effective treatment?
The available research shows that using steroids like prednisone and deflazacort in combination with exercise can improve muscle strength and function in patients with Duchenne Muscular Dystrophy. For example, one study found that boys treated with prednisone showed significant improvements in muscle strength and function within just one month, and these improvements peaked by three months. Another study highlighted that long-term use of corticosteroids can prolong the ability to walk by 2 to 5 years and improve heart and lung function. While there are side effects like weight gain and increased body hair, the benefits in muscle strength and quality of life are significant. Compared to no treatment, steroids help stabilize the condition and improve muscle strength, even if they don't significantly improve overall function.12345
What data supports the effectiveness of the drug Prednisone for Duchenne Muscular Dystrophy?
Research shows that Prednisone improves muscle strength and function in patients with Duchenne Muscular Dystrophy, with benefits seen as early as one month into treatment. Long-term use of corticosteroids like Prednisone can prolong walking ability, improve heart and lung function, and enhance quality of life.12345
What safety data exists for steroids and exercise in treating Duchenne Muscular Dystrophy?
Several studies have evaluated the safety of corticosteroids like prednisone and deflazacort in treating Duchenne Muscular Dystrophy (DMD). A trial with deflazacort showed improvements in muscle strength with side effects such as mild to moderate cushingoid appearance, increased appetite, body hair, irritability, and hyperactivity. Another study compared deflazacort and prednisone, noting that weight gain and behavioral side effects were more common with prednisone, while deflazacort had worse effects on bone health, growth, and cataracts. A large trial is also underway to standardize corticosteroid regimens and assess their safety profiles. Overall, while corticosteroids improve muscle function, they come with significant side effects that need careful management.16789
Is the combination of steroids and exercise safe for humans, particularly in the context of Duchenne Muscular Dystrophy?
Steroids like prednisone and deflazacort are generally safe for humans, but they can have side effects such as weight gain, increased appetite, irritability, and changes in appearance. Deflazacort may have fewer side effects compared to prednisone. Long-term use requires careful monitoring to manage these side effects.16789
Is the drug Prednisone a promising treatment for Duchenne Muscular Dystrophy?
How does the drug Prednisone differ from other treatments for Duchenne Muscular Dystrophy?
Prednisone is unique because it can quickly improve muscle strength in boys with Duchenne Muscular Dystrophy, with effects noticeable as early as 10 days after starting treatment. It is one of the few drugs that provide interim functional improvement while waiting for a definitive cure, and it can prolong walking ability and improve quality of life, although it may cause side effects like weight gain.45101112
What is the purpose of this trial?
This trial is testing if giving a smaller amount of medication less frequently, along with exercise, can help boys with a muscle condition. The current frequent treatment has bad side effects, but this new method might work just as well without them. The medication has been shown to improve muscle strength in boys with this condition, but its frequent use is associated with significant side effects.
Research Team
Tanja Taivassalo, MD
Principal Investigator
University of Florida
Eligibility Criteria
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.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive twice weekly prednisone for 12 months, with some participants also undergoing structured exercise training
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- In-home Exercise Training
- Prednisone
Prednisone is already approved in United States, European Union, Canada for the following indications:
- Allergic reactions
- Asthma
- Blood disorders
- Cancer
- Eye problems
- Immune system disorders
- Inflammatory conditions
- Multiple sclerosis
- Organ transplantation
- Rheumatoid arthritis
- Skin conditions
- Allergic reactions
- Asthma
- Blood disorders
- Cancer
- Eye problems
- Immune system disorders
- Inflammatory conditions
- Multiple sclerosis
- Organ transplantation
- Rheumatoid arthritis
- Skin conditions
- Allergic reactions
- Asthma
- Blood disorders
- Cancer
- Eye problems
- Immune system disorders
- Inflammatory conditions
- Multiple sclerosis
- Organ transplantation
- Rheumatoid arthritis
- Skin conditions
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Florida
Lead Sponsor
U.S. Army Medical Research and Development Command
Collaborator