What side effects are associated with this type of intervention?

Common treatments for Duchenne Muscular Dystrophy (DMD) include glucocorticoids, eteplirsen, and anti-inflammatory agents like Canakinumab. Glucocorticoids can cause weight gain, bone thinning, high blood pressure, and mood changes. Eteplirsen, an exon-skipping therapy, has been associated with increased dystrophin production but may lead to loss of ambulation in some patients. Canakinumab, an anti-IL1β antibody, is generally well-tolerated but can cause injection site reactions and increased risk of infections. Overall, while these treatments can offer benefits, they also come with potential side effects that need to be managed under medical supervision.

What prior FDA approvals exist?

The FDA has approved several treatments for Duchenne Muscular Dystrophy (DMD), focusing on different mechanisms of action. Eteplirsen, an exon-skipping drug, has been approved to increase dystrophin production in patients with specific genetic mutations. Other investigational therapies include gene therapy approaches using adeno-associated viral vectors to deliver microdystrophin genes. While Canakinumab, an anti-IL1β antibody, is being studied for its potential effects on muscle inflammation in DMD, it has not yet been approved for this indication. Current treatments aim to slow disease progression and improve muscle function.

What other types of research exist?

Apitegromab (SRK-015), an anti-promyostatin monoclonal antibody, is a promising novel alternative for Duchenne Muscular Dystrophy patients. It is currently under development to improve motor function in patients with spinal muscular atrophy, a condition with similarities to DMD.

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Monoclonal Antibodies

Canakinumab for Duchenne Muscular Dystrophy

Phase 1 & 2

Waitlist Available

Led By Christopher Spurney

Research Sponsored by Children's National Research Institute

Eligibility Criteria Checklist

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

Must have

Subject has a diagnosis of DMD

Subject is naïve to treatment with glucocorticoids for DMD

Must not have

Subject has current or history of chronic systemic fungal or viral infections

Subject received live vaccination within the previous month

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 4 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing canakinumab, a medication that reduces inflammation, in young boys with Duchenne Muscular Dystrophy (DMD) who have not been treated with steroids. The study aims to see if the medication is safe and effective by monitoring changes in blood markers over a short period. Canakinumab has been studied for various inflammatory conditions.

Who is the study for?

This trial is for young boys with Duchenne Muscular Dystrophy (DMD) who are between 2 to less than 6 years old, have not been treated with steroids, and can walk. They should have normal or acceptable lab test results and no major organ issues, diabetes, immunosuppression, recent infections or vaccinations. They must not be on other investigational drugs or have had certain treatments within the past 3 months.

What is being tested?

The trial tests Canakinumab Injection [Ilaris], an antibody against interleukin-1 beta (IL1β), for safety and potential efficacy in reducing muscle inflammation in DMD patients. Participants will receive a single subcutaneous dose of Canakinumab and will be monitored over 30 days for changes in serum biomarkers related to inflammation.

What are the potential side effects?

Potential side effects may include reactions at the injection site, increased risk of infection due to immune system suppression by the drug, abnormal blood test results reflecting liver or kidney function issues, allergic reactions including fever syndromes as seen in conditions where Canakinumab is already used.

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 Duchenne Muscular Dystrophy (DMD).

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I have not taken glucocorticoids for Duchenne Muscular Dystrophy.

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I can walk on my own.

Exclusion Criteria

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

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I have or had a long-term fungal or viral infection.

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I have received a live vaccine in the last month.

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I am under 2 years old.

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I have a history of major kidney, liver issues, diabetes, or a weak immune system.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 4 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~4 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 4 weeks for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Clinical adverse events

Laboratory adverse events

Secondary study objectives

Changes in serum biomarkers of inflammation after treatment

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: TreatmentExperimental Treatment1 Intervention

Canakinumab treatment

0 / 7Eligibility 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 glucocorticoids, exon-skipping drugs, and investigational therapies like gene therapy and anti-inflammatory agents such as Canakinumab. Glucocorticoids work by reducing inflammation and immune response, which helps to slow muscle degeneration. Exon-skipping drugs, such as eteplirsen and golodirsen, allow cells to skip over faulty parts of the dystrophin gene, enabling the production of a functional, albeit shorter, dystrophin protein. Gene therapy aims to introduce functional copies of the dystrophin gene into muscle cells. Canakinumab, an anti-IL1β antibody, targets inflammatory pathways by neutralizing interleukin-1 beta, potentially reducing muscle inflammation and damage. These treatments are crucial for DMD patients as they address the underlying genetic and inflammatory components of the disease, aiming to preserve muscle function and improve quality of life.

From diagnosis to therapy in Duchenne muscular dystrophy.