What side effects are associated with this type of intervention?

The most common treatments for Duchenne Muscular Dystrophy, particularly oral corticosteroids like prednisone and deflazacort, are associated with several side effects. These include growth retardation, Cushingoid appearance, elevated blood pressure, hyperglycemia, cataracts, and osteoporosis. Long-term use of glucocorticoids can also lead to significant adverse effects such as delayed disease progression and increased median age at loss of mobility milestones. It is important for patients and caregivers to discuss these potential side effects with their healthcare provider to manage and mitigate risks effectively.

What prior FDA approvals exist?

The FDA has approved glucocorticoids such as prednisone and deflazacort for the treatment of Duchenne Muscular Dystrophy (DMD). These drugs are known for their anti-inflammatory and immunosuppressive properties. Prednisone has been shown to improve muscle strength and motor function, as well as delay the progression of the disease. Deflazacort, preferred for its more favorable side effect profile, also improves motor function and delays loss of ambulation. Both drugs have demonstrated benefits in pulmonary function and reducing the risk of scoliosis in DMD patients.

What other types of research exist?

Promising novel alternatives to oral corticosteroids for Duchenne Muscular Dystrophy patients include deflazacort, which has been compared to prednisone in clinical trials, and other immunomodulatory agents such as mycophenolate mofetil, cyclosporine, and tacrolimus. These alternatives have shown potential in managing symptoms and slowing disease progression in various studies.

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Corticosteroid

Weekly Prednisolone for Duchenne Muscular Dystrophy

Phase 4

Recruiting

Led By Anne Connolly, MD

Research Sponsored by Anne M. Connolly

Eligibility Criteria Checklist

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

Must have

Subjects ages 1 month through 30 months

Weakness consistent with Duchenne on exam, creatine kinase ≥ 20 times the upper limit of normal, and genetic mutation known to be causative for DMD.

Must not have

Prior treatment with Glucocorticosteroids

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline visit to 24 month visit

Awards & highlights

All Individual Drugs Already Approved

Approved for 5 Other Conditions

Drug Has Already Been Approved

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing if a lower dose of steroids given once a week is as effective as a higher dose for infants and young boys with Duchenne muscular dystrophy. The goal is to see if the lower dose can reduce muscle damage while causing fewer side effects, like weight gain. The study will measure improvements in motor skills over time. Prednisolone has been shown to improve strength in Duchenne dystrophy, but high doses are associated with significant side effects.

Who is the study for?

This trial is for young children from 1 month to 30 months old who show signs of Duchenne Muscular Dystrophy (DMD) on physical exams, have high levels of an enzyme called creatine kinase in their blood, and a genetic mutation known to cause DMD. Children previously treated with glucocorticosteroids can't participate.

What is being tested?

The study is testing if a lower weekly dose of the corticosteroid Prednisolone (5mg/kg) is as effective as the standard higher dose (10mg/kg) for treating symptoms of Duchenne Muscular Dystrophy in infants.

What are the potential side effects?

Prednisolone may cause side effects such as increased appetite, weight gain, mood changes, high blood pressure, bone weakening over time, and higher risk of infections.

Eligibility Criteria

Inclusion Criteria

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

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I am between 1 month and 30 months old.

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I have Duchenne muscular dystrophy with high creatine kinase levels and a confirmed genetic mutation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been treated with steroids before.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline visit to 24 month visit

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline visit to 24 month visit

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline visit to 24 month visit for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

The change from baseline to 24 months for the Gross Motor Scaled Score.

Secondary study objectives

Language (expressive and receptive), Social and Fine Motor skills at 24 months as assessed by the Bayley-4 Scales of Infant and Toddler Development

Linear growth

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: ExperimentalExperimental Treatment1 Intervention

This is a one-arm study and the group of subjects are all experimental and will receive drug.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Prednisolone

FDA approved

0 / 3Eligibility 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

The most common treatments for Duchenne Muscular Dystrophy (DMD) include oral corticosteroids and genetic therapies. Oral corticosteroids, such as prednisone, work by reducing inflammation and suppressing the immune system, which helps to slow muscle degeneration and improve muscle strength. This is crucial for DMD patients as it can prolong mobility and improve quality of life. Genetic therapies, like exon skipping drugs (eteplirsen, golodirsen, viltolarsen), aim to increase the production of dystrophin, a protein that is deficient in DMD patients. By skipping over specific exons in the DMD gene, these therapies allow for the production of a shorter but functional dystrophin protein, which can help stabilize muscle fibers and slow disease progression. Both treatment approaches are vital as they address the underlying mechanisms of muscle deterioration in DMD.