Dupilumab for Preventing Asthma Attacks in Children

(PANDA Trial)

Recruiting at 7 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Must be taking: Fluticasone, LABA

Must not be taking: Immunotherapy, Biologic therapy

Disqualifiers: Pregnancy, Smoking, Cancer, others

Stay on Your Current Meds You can continue your current medications while participating

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: 4

Duration: 12 months

240 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a multi-center, double-blind, placebo-controlled, randomized trial of dupilumab adjunctive therapy for prevention of asthma exacerbations in urban children and adolescents with T2-high exacerbation-prone asthma.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but it does require that your asthma medications be managed by the study clinician. You may need to adjust your asthma medications to follow the study protocol.

What data supports the effectiveness of the drug Dupilumab for preventing asthma attacks in children?

Dupilumab has been shown to reduce severe asthma attacks and improve lung function in patients aged 12 and older with moderate-to-severe asthma, especially those with type 2 inflammation. It is already approved for use in adults and adolescents for asthma and other conditions like atopic dermatitis, indicating its potential effectiveness. ^{1 2 3 4 5}

Is dupilumab generally safe for use in humans?

Dupilumab is generally considered safe for humans, with studies showing minimal adverse events in patients with moderate to severe asthma. However, some ocular (eye-related) adverse events have been reported, mainly in patients treated for atopic dermatitis. ^{2 5 6 7 8}

How is the drug dupilumab different from other asthma treatments?

Dupilumab is unique because it targets the IL-4 and IL-13 signaling pathways, which are key drivers of type 2 inflammation in asthma. This makes it effective for both eosinophilic and non-eosinophilic severe asthma, offering an advantage over other biologics that may not address both types. ^{2 3 4 5 9}

Research Team

Daniel J. Jackson, M.D.

Principal Investigator

University of Wisconsin School of Medicine and Public Health; Division of Allergy & Immunology

Eligibility Criteria

This trial is for urban children and adolescents aged 6-17 with T2-high exacerbation-prone asthma. They must have had at least two asthma attacks in the past year, be on certain asthma medications, and not live in specific areas or have public health insurance. Participants need a diagnosis of asthma made over a year ago, meet certain blood test criteria, and can perform spirometry tests.

Inclusion Criteria

I am 12 or older and use a specific asthma inhaler or its equivalent.

I am aged 6-11 and use a strong asthma inhaler or similar treatment.

You have high levels of eosinophils in your blood or FeNO in your breath.

Exclusion Criteria

I am not pregnant, breastfeeding, nor planning to become pregnant and will use birth control during the study.

I do not have an active parasitic infection or am not currently being treated for one.

You have a known condition that weakens your immune system.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Run-in

Participants undergo a 4-week run-in period to ensure continued eligibility

4 weeks

1 visit (in-person)

Treatment

Participants receive dupilumab or placebo injections every 2 or 4 weeks for 12 months

12 months

Injections every 2 or 4 weeks, E&M visits every 3 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months

Regular follow-up visits

Treatment Details

Interventions

• Dupilumab

Trial Overview The study is testing Dupilumab as an additional treatment to prevent asthma flare-ups compared to a placebo (a substance with no therapeutic effect). It's conducted across multiple centers where participants are randomly assigned to either receive Dupilumab or placebo without knowing which one they're getting.

Participant Groups

Group I: Dupilumab Experimental Treatment 1 Intervention

Participants between 12-17 years of age will receive an initial dose of 600 mg (two 300 mg injections) followed by 300 mg given every other week (Q2W). Participants between 6-11 years of age will not complete a loading dose and will receive injections based on their body weight: Those weighing 15 kg to less than 30 kg will receive 300 mg every four weeks (Q4W). Participants in this weight category who were randomized before July 1, 2024, were assigned to a 100mg Q2W and will not be transitioned to the Q4W dosing schedule. Those with a body weight of 30 kg or more will receive 200 mg Q2W.

Group II: Placebo Placebo Group 1 Intervention

Participants between 12-17 years of age will receive an initial dose of placebo (two injections) followed by a placebo injection given every other week (Q2W). Participants between 6-11 years of age will not receive an initial loading dose of placebo and will receive injections Q2W or Q4W based on their body weight and date of randomization.: The injection volume of placebo will be matched to the corresponding dupilumab dose based on participant body weight.

Who Is Running the Clinical Trial?

National Institute of Allergy and Infectious Diseases (NIAID)

Lead Sponsor

Trials

3,361

Recruited

5,516,000+

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

Rho Federal Systems Division, Inc.

Industry Sponsor

Trials

44

Recruited

15,000+

Childhood Asthma in Urban Settings (CAUSE)

Collaborator

Trials

1

Recruited

240+

Findings from Research

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]

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 is a monoclonal antibody that targets the IL-4 receptor, effectively reducing severe asthma exacerbations and improving lung function in patients aged 12 and older with moderate-to-severe asthma, based on three pivotal trials lasting 24-52 weeks.

The treatment is generally well tolerated and allows for a reduction in oral corticosteroid doses without compromising asthma control, particularly benefiting patients with higher levels of type 2 inflammation, such as those with elevated eosinophils.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dupilumab: A Review in Moderate to Severe Asthma. Deeks, ED.[2020]

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, 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]

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]

In the Phase 3 LIBERTY ASTHMA QUEST study, dupilumab significantly reduced severe asthma exacerbation rates by up to 67% compared to placebo in patients with elevated eosinophils, regardless of their inhaled corticosteroid dose or baseline lung function.

Dupilumab also improved lung function (measured by FEV1), asthma control, quality of life, and reduced levels of fractional exhaled nitric oxide (FeNO) across all patient subgroups, demonstrating its efficacy in managing moderate-to-severe asthma.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dupilumab Efficacy in Patients Stratified by Baseline Treatment Intensity and Lung Function. Pavord, ID., Siddiqui, S., Papi, A., et al.[2022]

Dupilumab is a monoclonal antibody that blocks IL-4 and IL-13 signaling, which are key drivers of atopic diseases, and it was first approved by the FDA in March 2017 for treating moderate-to-severe atopic dermatitis in adults who do not respond to topical treatments.

In October 2018, dupilumab was also approved as an add-on therapy for moderate-to-severe asthma in patients aged 12 and older, particularly those with eosinophilic asthma or those dependent on oral corticosteroids, highlighting its efficacy in managing multiple atopic conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A review of dupilumab in the treatment of atopic diseases. Thibodeaux, Q., Smith, MP., Ly, K., et al.[2020]

References

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

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

2. England pubmed.ncbi.nlm.nih.gov

Dupilumab for the treatment of asthma. [2019]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Dupilumab: A Review in Moderate to Severe Asthma. [2020]

4. 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]

5. England pubmed.ncbi.nlm.nih.gov

Dupilumab in the treatment of asthma. [2020]

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. New Zealand pubmed.ncbi.nlm.nih.gov

Dupilumab Efficacy in Patients Stratified by Baseline Treatment Intensity and Lung Function. [2022]

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

A review of dupilumab in the treatment of atopic diseases. [2020]