89 Participants Needed

Steroids + Exercise for Duchenne Muscular Dystrophy

TT
Overseen ByTanja Taivassalo, PhD
Age: < 18
Sex: Male
Trial Phase: Phase 2
Sponsor: University of Florida
Must be taking: Glucocorticoids
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 3 JurisdictionsThis treatment is already approved in other countries

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?

Yes, Prednisone is a promising drug for Duchenne Muscular Dystrophy. It improves muscle strength and function, helps patients walk longer, reduces the need for surgeries, and enhances heart and lung health. It also increases survival and quality of life.45101112

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

TT

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

I have been diagnosed with DMD based on tests and physical exams.
I am between 5 and 9 years old.
I have been on a stable daily medication regimen for 6 months.
See 2 more

Exclusion Criteria

Contraindication to an MR examination
I do not have unstable health issues like heart problems.
I have a condition that affects my muscle function or metabolism.
See 4 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive twice weekly prednisone for 12 months, with some participants also undergoing structured exercise training

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • In-home Exercise Training
  • Prednisone
Trial Overview 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.
Participant Groups
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.

Prednisone is already approved in United States, European Union, Canada for the following indications:

🇺🇸
Approved in United States as Prednisone for:
  • Allergic reactions
  • Asthma
  • Blood disorders
  • Cancer
  • Eye problems
  • Immune system disorders
  • Inflammatory conditions
  • Multiple sclerosis
  • Organ transplantation
  • Rheumatoid arthritis
  • Skin conditions
🇪🇺
Approved in European Union as Prednisone for:
  • Allergic reactions
  • Asthma
  • Blood disorders
  • Cancer
  • Eye problems
  • Immune system disorders
  • Inflammatory conditions
  • Multiple sclerosis
  • Organ transplantation
  • Rheumatoid arthritis
  • Skin conditions
🇨🇦
Approved in Canada as Prednisone for:
  • 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

Trials
1,428
Recruited
987,000+

U.S. Army Medical Research and Development Command

Collaborator

Trials
296
Recruited
249,000+

Findings from Research

In a double-blind trial involving 28 patients with Duchenne muscular dystrophy, Deflazacort (DF) showed statistically significant improvements in muscle strength and function compared to a placebo, indicating its efficacy as a treatment option.
While some mild side effects were observed, such as increased appetite and a cushingoid appearance in a few patients, no one had to stop taking the medication, suggesting that DF may have a better safety profile than traditional steroids like prednisone.
Steroids in Duchenne muscular dystrophy--deflazacort trial.Mesa, LE., Dubrovsky, AL., Corderi, J., et al.[2019]
In a six-month trial involving 103 boys with Duchenne's muscular dystrophy, both doses of prednisone (0.75 mg/kg and 1.5 mg/kg) significantly improved muscle strength and function compared to placebo, with peak improvements observed by three months.
Despite the benefits in muscle function, some patients still required mobility aids, and side effects like weight gain and excessive hair growth were noted, indicating the need for further research on long-term corticosteroid use and its mechanisms.
Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy.Mendell, JR., Moxley, RT., Griggs, RC., et al.[2022]
In a study of 20 pediatric patients with Duchenne muscular dystrophy, those who received corticosteroid treatment (deflazacort) experienced stabilization in muscular strength and functional performance, while untreated patients showed progressive worsening.
Corticosteroid treatment led to improved muscular balance in 70% of patients, although only 2% showed significant functional improvement, indicating that while steroids can help stabilize the disease, they may not prevent loss of independent gait, which occurred at similar ages in both treated and untreated groups.
[Effects of corticosteroids in the management of Duchenne muscular dystrophy: our experience].Rafia, S., Pascual-Pascual, SI., Martínez-Granero, MA., et al.[2018]

References

Steroids in Duchenne muscular dystrophy--deflazacort trial. [2019]
Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. [2022]
[Effects of corticosteroids in the management of Duchenne muscular dystrophy: our experience]. [2018]
Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group. [2019]
Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment: implications for management. [2022]
Deflazacort treatment of Duchenne muscular dystrophy. [2014]
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy. [2018]
Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy. [2022]
Safety and clinical outcome of tamoxifen in Duchenne muscular dystrophy. [2022]
10.United Statespubmed.ncbi.nlm.nih.gov
Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months) [2019]
11.United Statespubmed.ncbi.nlm.nih.gov
Corticosteroids in Duchenne muscular dystrophy: a reappraisal. [2017]
12.United Statespubmed.ncbi.nlm.nih.gov
The role of corticosteroids in muscular dystrophy: a critical appraisal. [2022]
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