Dupilumab for Asthma

Recruiting in Cincinnati (>99 mi)

+105 other locations

Overseen By Trial Transparency email recommended (Toll free for US & Canada)

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Sanofi

Must be taking: Inhaled corticosteroids

Must not be taking: Oral corticosteroids

Disqualifiers: Severe asthma, Anaphylaxis, Prematurity, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

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: Unknown

Duration: 52 weeks

90 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a parallel, Phase 3, 2-arm study to evaluate the efficacy and long-term safety of dupilumab treatment in children 2 to \<6 years of age with uncontrolled asthma and/or recurrent severe asthmatic wheeze. The study will be conducted in 2 parts. Part A will be a 52-week, randomized, double-blind, placebo-controlled study to assess the safety and efficacy of dupilumab in children aged 2 to \<6 years old with uncontrolled asthma and/or recurrent severe asthmatic wheeze. At the end of Part A, all eligible participants will be offered participation in Part B, an optional open-label extension phase. Study details include: Part A: The study duration of part A will be up to 68 weeks consisting of a 4-week Screening, a 52week treatment period, and a 12-week post-treatment follow-up period. For participants who will chose to participate in Part B, the study duration will be up to 120 weeks (additional 52-week treatment period). Part B: For participants who will choose to participate in Part B, the study duration will be up to 120 weeks (Part A \[4-week Screening and a 52-week treatment period\] plus additional 52-week treatment period and a 12-week post-treatment follow-up period).

Do I need to stop my current medications to join the trial?

The trial does not specify if you need to stop taking your current medications. However, it mentions that participants must have been using a stable dose of inhaled corticosteroids (ICS) for at least one month before the screening, which suggests you may need to continue your current asthma medication.

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 as well. ^{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. ^{3 4 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 the inflammation process of asthma, and it may be effective for both eosinophilic and non-eosinophilic severe asthma types, unlike some other treatments that target only one type. ^{1 2 3 4 7}

Eligibility Criteria

This trial is for children aged 2 to less than 6 with uncontrolled asthma or severe asthmatic wheeze, diagnosed with Atopic Dermatitis. They must have been on inhaled corticosteroids for at least a month and show allergic reactions to certain foods or airborne allergens. Parents need to understand study requirements and help their kids follow the protocol.

Inclusion Criteria

Parents or guardians are able to bring the child to all clinic visits and follow study instructions.

The parent(s) or guardian(s) should be able to understand what is needed for the study.

Participants/parent(s)/caregiver(s)/legal guardian(s) must be able to understand and complete study-related questionnaires

Exclusion Criteria

I have had severe asthma attacks that were life-threatening.

I have severe asthma and need to take steroids regularly.

History of a systemic hypersensitivity reaction or anaphylaxis to dupilumab or any other biologic therapy

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Treatment

Participants receive either dupilumab or placebo for 52 weeks in a double-blind manner

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment with dupilumab for an additional 52 weeks

52 weeks

Treatment Details

Interventions

• Dupilumab

Trial Overview The study tests Dupilumab's effectiveness and safety against a placebo over a year (Part A), followed by an optional second year (Part B). It's double-blind, meaning neither participants nor researchers know who gets the real drug during Part A.

Participant Groups

Group I: Dupilumab (double-blind period) Experimental Treatment 1 Intervention

Dupilumab subcutaneous injection as per protocol

Group II: Placebo Placebo Group 1 Intervention

Placebo matching dupilumab subcutaneous injection as per protocol

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

Approved in United States as Dupixent for:

• Atopic dermatitis
• Asthma
• Chronic rhinosinusitis with nasal polyps
• Eosinophilic esophagitis

Approved in European Union as Dupixent for:

• Atopic dermatitis
• Asthma
• Chronic rhinosinusitis with nasal polyps
• Eosinophilic esophagitis

Who Is Running the Clinical Trial?

Sanofi

Lead 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

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

Findings from Research

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]

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 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]

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]

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]

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]

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]

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

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

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

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

3. 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]

4. England pubmed.ncbi.nlm.nih.gov

Dupilumab for the treatment of asthma. [2019]

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

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

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

Dupilumab in the treatment of asthma. [2020]