Canakinumab for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

Overseen byChristopher Spurney

Age: Any Age

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Children's National Research Institute

No Placebo Group

Breakthrough Therapy

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

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.

Research Team

Christopher Spurney

Principal Investigator

Children's National Research Institute

Eligibility Criteria

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.

Inclusion Criteria

I am at least 2 years old.

Your lab test results are mostly normal, and if not, they are not too far from normal. You also tested negative for tuberculosis.

I have been diagnosed with Duchenne Muscular Dystrophy (DMD).

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Exclusion Criteria

I have used or am using oral steroids or other drugs that weaken my immune system.

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

Subject has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator

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Treatment Details

Interventions

Canakinumab (Monoclonal Antibodies)

Trial OverviewThe 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.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: TreatmentExperimental Treatment1 Intervention

Canakinumab treatment