Canakinumab for Duchenne Muscular Dystrophy

Not currently recruiting at 1 trial location

Overseen By Christopher Spurney, MD

Age: Any Age

Sex: Male

Trial Phase: Phase 1 & 2

Sponsor: Children's National Research Institute

Must not be taking: Glucocorticoids, Immunosuppressants

Disqualifiers: Renal impairment, Hepatic impairment, Diabetes, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 4

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effects of canakinumab, a medication, on young boys with Duchenne Muscular Dystrophy (DMD), a condition that causes muscle weakness. The researchers aim to determine if this treatment is safe and affects specific blood markers indicating muscle inflammation. Participants will receive a single dose of canakinumab and will be monitored for a month. Boys diagnosed with DMD who can walk and have not received steroid treatment for DMD may be suitable for this study. As a Phase 1, Phase 2 trial, this research seeks to understand how the treatment works in people and measure its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking research.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are taking oral glucocorticoids or other immunosuppressive agents, you may not be eligible unless they were used for a different condition and stopped at least 3 months before the trial. Inhaled or topical glucocorticoids are allowed if the dose has been stable for at least 3 months.

Is there any evidence suggesting that canakinumab is likely to be safe for humans?

Research has shown that canakinumab has been tested in children with Duchenne muscular dystrophy (DMD) and is considered safe. In earlier studies, children received a dose of 2 mg per kilogram of body weight, which they tolerated well. These studies also found encouraging changes in biomarkers, indicating how the drug might work in the body. Canakinumab is already approved for other conditions in children, suggesting it is generally safe for young patients. While canakinumab is still under investigation for DMD, existing evidence supports its safe use in children. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Duchenne Muscular Dystrophy?

Canakinumab is unique because it offers a new approach to treating Duchenne Muscular Dystrophy (DMD) by targeting inflammation. Unlike the standard treatments like corticosteroids that broadly suppress the immune system, Canakinumab specifically blocks a protein called interleukin-1 beta, which is involved in inflammation. This targeted action could potentially reduce muscle damage more effectively while minimizing side effects. Researchers are excited because Canakinumab's precise mechanism might improve muscle function and slow disease progression in a way that current therapies can't.

What evidence suggests that canakinumab might be an effective treatment for Duchenne Muscular Dystrophy?

Research has shown that canakinumab, the investigational treatment in this trial, might help treat inflammatory conditions like Duchenne Muscular Dystrophy (DMD). Studies have found that a dose of 2 mg/kg is safe for children with DMD and may help reduce muscle inflammation. Canakinumab already treats other inflammatory conditions, such as systemic juvenile idiopathic arthritis, supporting its potential use for DMD. These early results suggest canakinumab could help manage muscle inflammation in DMD, but more research is needed to confirm its long-term benefits. ^{1 2 3 6 7}

Who Is on the Research Team?

Christopher Spurney

Principal Investigator

Children's National Research Institute

Are You a Good Fit for This Trial?

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).

Exclusion Criteria

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

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

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

1 visit (in-person)

Treatment

Participants receive a single subcutaneous dose of canakinumab

1 day

1 visit (in-person)

Post-treatment Evaluation

Participants undergo safety and serum biomarker monitoring

30 days

2 visits (in-person), 1 phone assessment

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Canakinumab

Trial Overview

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.

How Is the Trial Designed?

Group I: Treatment Experimental Treatment 1 Intervention

Canakinumab treatment

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

Approved in European Union as Ilaris for:

• Cryopyrin-Associated Periodic Syndromes (CAPS)
• Familial Cold Autoinflammatory Syndrome (FCAS)
• Muckle-Wells Syndrome (MWS)
• Tumor Necrosis Factor Receptor-Associated Periodic Syndrome (TRAPS)
• Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD)
• Familial Mediterranean Fever (FMF)
• Systemic Juvenile Idiopathic Arthritis (SJIA)
• Adult-Onset Still's Disease (AOSD)

Approved in United States as Ilaris for:

• Cryopyrin-Associated Periodic Syndromes (CAPS)
• Familial Cold Autoinflammatory Syndrome (FCAS)
• Muckle-Wells Syndrome (MWS)
• Tumor Necrosis Factor Receptor-Associated Periodic Syndrome (TRAPS)
• Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD)
• Familial Mediterranean Fever (FMF)
• Systemic Juvenile Idiopathic Arthritis (SJIA)
• Adult-Onset Still's Disease (AOSD)

Who Is Running the Clinical Trial?

Children's National Research Institute

Lead Sponsor

Trials

227

Recruited

258,000+

Foundation to Eradicate Duchenne

Collaborator

Trials

1

Recruited

3+

Published Research Related to This Trial

In a phase 2 trial involving 120 boys with Duchenne muscular dystrophy (DMD), domagrozumab was found to be generally safe and well tolerated, with most adverse events being mild and not related to the treatment.

However, the study did not show a significant treatment effect on functional measures, such as the 4-stair climb time, indicating that domagrozumab may not provide the expected benefits in improving muscle function in DMD patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy. Wagner, KR., Abdel-Hamid, HZ., Mah, JK., et al.[2021]

Canakinumab demonstrated long-term efficacy in treating active systemic juvenile idiopathic arthritis (JIA), with 62% of patients achieving a 50% improvement in disease activity after 2 years, and sustained efficacy observed for up to 5 years.

The treatment was associated with a significant reduction in glucocorticoid use, with 15.6% of patients discontinuing and 22% tapering their dosage, while no new safety concerns were identified during the long-term study.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Canakinumab in patients with systemic juvenile idiopathic arthritis and active systemic features: results from the 5-year long-term extension of the phase III pivotal trials. Ruperto, N., Brunner, HI., Quartier, P., et al.[2019]

Citations

1.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/40396427/

Pilot study of canakinumab (Ilaris) in steroid naïve children ...

Canakinumab 2 mg/kg dose is safe for children with DMD and demonstrated potential response biomarkers of efficacy in treating related muscle disease.

2.

parentprojectmd.org

parentprojectmd.org/clinical-trial/single-escalating-dose-pilot-trial-of-canakinumab-ilaris-in-duchenne-muscular-dystrophy/

Single Escalating Dose Pilot Trial of Canakinumab (ILARIS ...

The study is an open-label, single ascending dose study to assess safety and to evaluate short-term changes in biomarkers. The first 3 boys will receive ...

3.

withpower.com

withpower.com/trial/phase-2-muscular-dystrophy-duchenne-4-2019-ea5bd

Canakinumab for Duchenne Muscular Dystrophy

Canakinumab has shown effectiveness in treating other inflammatory conditions like cryopyrin-associated periodic syndromes (CAPS), where it provided rapid and ...

4.

journals.sagepub.com

journals.sagepub.com/doi/10.1177/22143602251319177

Pilot study of canakinumab (Ilaris) in steroid naïve children ...

We completed an open-label, single dose pilot study of canakinumab 2 mg/kg subcutaneous injection in steroid naïve boys with DMD older than 2 years of age to ...

5.

orb.binghamton.edu

orb.binghamton.edu/cgi/viewcontent.cgi?article=1076&context=pharmacy_fac

Pilot study of canakinumab (Ilaris) in steroid naïve children ...

Further studies using canakinumab for a longer treatment period may demonstrate increased benefit. Keywords. Duchenne muscular dystrophy, ...

6.

ctv.veeva.com

ctv.veeva.com/study/single-escalating-dose-pilot-trial-of-canakinumab-ilaris-r-in-duchenne-muscular-dystrophy

Single Escalating Dose Pilot Trial of Canakinumab (ILARIS ...

This study is a pilot trial to investigate the effects of canakinumab on clinical safety and potential clinical efficacy as demonstrated by ...

7.

parentprojectmd.org

parentprojectmd.org/drug-development-pipeline/canakinumab-ilaris/

Canakinumab (ILARIS®)

Canakinumab is a moloconal antibody, targeting IL1b, used to treat auto-inflammatory syndromes in children. Canakinumab may reduce inflammation in Duchenne.