Dupilumab for Asthma

(IDEA Trial)

Recruiting at6 trial locations

Overseen by Wanda Phipatanakul, MD. MSc.

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Recruiting

Sponsor: Boston Children's Hospital

Must be taking: Inhaled corticosteroids, Biologics

Must not be taking: Immunosuppressives, Immunomodulators

Disqualifiers: Chronic lung disease, Smoking, Pregnancy, others

Prior Safety Data

Breakthrough Therapy

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

The Goal of this study is to investigate if individuals ages 12 years and older, carrying the IL-4RαR576 gene variant, will have a greater response to therapy acting directly on the anti-IL-4R. This will be conducted by examining the effect of a 48 week therapy with dupilumab on the rate of asthma exacerbations.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications. However, if you are on allergen immunotherapy, you must continue it for the duration of the study.

What data supports the effectiveness of the drug Dupilumab for asthma?

Dupilumab has shown effectiveness in treating severe type-2 asthma by targeting specific pathways involved in inflammation, and its efficacy has been demonstrated in clinical trials. It is already used for other conditions like atopic dermatitis and chronic rhinitis, indicating its potential in managing asthma symptoms. ^{1 2 3 4 5}

Is dupilumab safe for treating asthma?

Dupilumab is generally considered safe for treating moderate to severe asthma, with studies showing minimal adverse events compared to a placebo. However, it may cause an increase in blood eosinophils (a type of white blood cell) and has been associated with some eye-related side effects, mainly in patients treated for atopic dermatitis. ^{1 2 6 7 8}

How is the drug Dupilumab unique for treating asthma?

Dupilumab is unique because it targets the IL-4 and IL-13 signaling pathways, which are involved in type 2 inflammation, and it may be effective for both eosinophilic and non-eosinophilic severe asthma, unlike some other treatments that only target one type. It is also used for other conditions like atopic dermatitis, showing its versatility. ^{1 2 3 4 8}

Research Team

Wanda Phipatanakul, MD. MSc.

Principal Investigator

Boston Children's Hospital

Eligibility Criteria

This trial is for individuals aged 12 and older with asthma, who can perform lung function tests and have had an asthma attack in the past year. They must be on a stable dose of corticosteroids or have used biologic medication recently. Smokers or recent quitters, pregnant/breastfeeding women, those with other chronic lung diseases, or using certain drugs are excluded.

Inclusion Criteria

Ability to provide informed consent

I can do breathing tests.

I am a woman who can have children and my pregnancy test is negative.

Exclusion Criteria

Evidence that the participant or family may be unreliable or poorly adherent to their asthma treatment or study procedures

I am not currently on immunosuppressants for conditions other than asthma.

You are currently smoking or quit smoking less than 6 months ago.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-12 weeks

Run-in

Run-in period to determine asthma control before randomization

2-12 weeks

Treatment

Participants receive subcutaneous Dupilumab or placebo every two weeks for 48 weeks

48 weeks

Bi-weekly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Dupilumab (Monoclonal Antibodies)

Trial Overview The study examines if Dupilumab reduces asthma attacks over 48 weeks in patients carrying the IL-4RαR576 gene variant compared to a placebo. Participants' response to this anti-IL-4R therapy will be monitored by tracking exacerbation rates.

Participant Groups

Group I: Treatment group Experimental Treatment 1 Intervention

Dupilumab (Dupixent®) administered subcutaneously every two weeks. An initial dose of 600 mg (two 300 mg injections) followed by 300 mg given every other week.

Group II: Placebo group Placebo Group 1 Intervention

Placebo (preparation, administration, packaging, and labeling all equivalent to the treatment) administered subcutaneously every two weeks.

Who Is Running the Clinical Trial?

Boston Children's Hospital

Lead Sponsor

Trials

801

Recruited

5,584,000+

Dr. Kevin B. Churchwell

Boston Children's Hospital

Chief Executive Officer since 2021

MD from Vanderbilt Medical School

Dr. Sarah Pitts

Boston Children's Hospital

Chief Medical Officer since 2019

MD from Harvard Medical School

Sanofi

Industry Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

National Institute of Allergy and Infectious Diseases (NIAID)

Collaborator

Trials

3,361

Recruited

5,516,000+

Dr. Jeanne Marrazzo

National Institute of Allergy and Infectious Diseases (NIAID)

Chief Executive Officer since 2023

MD, MPH

Dr. H. Clifford Lane

National Institute of Allergy and Infectious Diseases (NIAID)

Chief Medical Officer

MD

Regeneron Pharmaceuticals

Industry Sponsor

Trials

690

Recruited

948,000+

Founded

1988

Headquarters

Tarrytown, USA

Known For

Precision medicine

Top Products

Dupixent, EYLEA, Libtayo, Praluent

Leonard Schleifer

Regeneron Pharmaceuticals

Chief Executive Officer since 1988

MD and PhD in Medicine

George Yancopoulos

Regeneron Pharmaceuticals

Chief Medical Officer since 1997

MD from Harvard Medical School

HealthBeacon Plc

Industry Sponsor

Trials

2

Recruited

160+

Merck Sharp & Dohme LLC

Industry Sponsor

Trials

4,096

Recruited

5,232,000+

Chirfi Guindo

Merck Sharp & Dohme LLC

Chief Marketing Officer since 2022

Degree in Engineering from Ecole Centrale de Paris, MBA from New York University Stern School of Business

Robert M. Davis

Merck Sharp & Dohme LLC

Chief Executive Officer since 2021

JD from Northwestern University Pritzker School of Law, MBA from Northwestern University Kellogg Graduate School of Management, Bachelor's in Finance from Miami University

Findings from Research

Dupilumab is a monoclonal antibody that targets the IL-4 receptor, blocking signals from IL-4 and IL-13, and is currently in phase III trials for treating moderate-to-severe asthma, showing promise for both eosinophilic and non-eosinophilic asthma phenotypes.

While dupilumab appears to be generally well tolerated, further large-scale studies are necessary to fully evaluate its long-term safety and efficacy, especially in pediatric populations where data is currently lacking.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dupilumab for the treatment of asthma. Santini, G., Mores, N., Malerba, M., et al.[2019]

In a real-life study of 12 patients with severe type-2 asthma, dupilumab treatment for 3 months led to significant improvements in asthma control (ACT score) and lung function (FEV1), indicating its efficacy in managing symptoms.

The treatment also resulted in a notable reduction in FeNO levels, a biomarker for type-2 inflammation, although blood eosinophil counts did not show significant change, suggesting that dupilumab effectively targets the IL4/IL13 pathway in asthma management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Early effectiveness of type-2 severe asthma treatment with dupilumab in a real-life setting; a FeNO-driven choice that leads to winning management. Carpagnano, GE., Scioscia, G., Buonamico, E., et al.[2022]

Dupixent (dupilumab) is set to receive FDA approval for asthma treatment on October 20, expanding its use beyond atopic dermatitis.

Once approved, Dupixent will be part of a group of monoclonal antibodies, including Nucala, Cinqair, and Fasenra, that target type 2 inflammation in severe asthma, highlighting its role in managing this specific asthma phenotype.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dupixent, a New Entrant In the Asthma Lists. Reinke, T.[2019]

In a study of 99 patients with severe asthma, the majority exhibited diverse clinical characteristics, with 58% having adult-onset asthma and 48% showing an allergic phenotype, indicating a range of profiles among those eligible for dupilumab treatment.

The analysis revealed that 70% of patients met at least one GINA criterion for type 2 airway inflammation, and 64% had comorbidities associated with type 2 inflammation, highlighting the complexity of managing severe asthma in real-world settings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-World Characteristics of Patients with Severe Asthma prior to Starting Dupilumab: The ProVENT Study. Korn, S., Schmidt, O., Timmermann, H., et al.[2023]

Dupilumab significantly improved the quality of life for adolescents with uncontrolled severe asthma within just 4 weeks of treatment, with 80% of participants able to reduce their inhaled corticosteroid dosage.

Throughout the 24-week study, none of the five adolescents experienced asthma exacerbations, indicating that dupilumab effectively controlled their asthma symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Time effect of dupilumab to treat severe uncontrolled asthma in adolescents: A pilot study. Indolfi, C., Dinardo, G., Klain, A., et al.[2023]

Dupilumab significantly improves lung function and asthma control in patients with moderate to severe asthma, as evidenced by better forced expiratory volume and asthma control questionnaire scores over 12 and 24 weeks in 13 trials.

The treatment is generally safe, showing no significant increase in adverse events compared to placebo, although it was associated with higher blood eosinophil levels.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dupilumab efficacy and safety in patients with moderate to severe asthma: A systematic review and meta-analysis. Zaazouee, MS., Alwarraqi, AG., Mohammed, YA., et al.[2022]

Dupilumab, a monoclonal antibody that blocks IL-4 and IL-13 signaling, has been shown to significantly improve asthma symptoms and reduce exacerbation rates in adolescents and adults, based on Phase II and III clinical trials.

The treatment has a favorable safety profile and has also been approved for use in atopic dermatitis, highlighting its effectiveness across multiple atopic diseases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dupilumab in the treatment of asthma. Grey, A., Katelaris, CH.[2020]

In a study of 46 adult patients with moderate-to-severe atopic dermatitis treated with dupilumab, 34.8% experienced ocular adverse events (OAEs), primarily mild to moderate dry eye disease and superficial punctate keratitis.

Patients with pre-existing conditions such as dry eye disease, eyelid eczema, or high IgE levels were more likely to develop OAEs, suggesting that these factors should be considered before starting dupilumab treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Incidence and risk factors for dupilumab associated ocular adverse events: a real-life prospective study. Touhouche, AT., Cassagne, M., Bérard, E., et al.[2021]

References

1. England pubmed.ncbi.nlm.nih.gov

Dupilumab for the treatment of asthma. [2019]

2. Italy pubmed.ncbi.nlm.nih.gov

Early effectiveness of type-2 severe asthma treatment with dupilumab in a real-life setting; a FeNO-driven choice that leads to winning management. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Dupixent, a New Entrant In the Asthma Lists. [2019]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Real-World Characteristics of Patients with Severe Asthma prior to Starting Dupilumab: The ProVENT Study. [2023]

5. Singapore pubmed.ncbi.nlm.nih.gov

Time effect of dupilumab to treat severe uncontrolled asthma in adolescents: A pilot study. [2023]

6. Switzerland pubmed.ncbi.nlm.nih.gov

Dupilumab efficacy and safety in patients with moderate to severe asthma: A systematic review and meta-analysis. [2022]

7. England pubmed.ncbi.nlm.nih.gov

Dupilumab in the treatment of asthma. [2020]

8. England pubmed.ncbi.nlm.nih.gov

Incidence and risk factors for dupilumab associated ocular adverse events: a real-life prospective study. [2021]