Dupilumab for Preventing Asthma Attacks in Children

(PANDA Trial)

Recruiting at 9 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether the drug dupilumab (also known as Dupixent) can prevent asthma attacks in children and teens living in urban areas. The study compares dupilumab to a placebo (a non-active substance) to determine if it reduces asthma flare-ups. Children and teens who have had asthma for over a year and experienced at least two severe asthma attacks in the past year might be suitable for this trial. Participants must live in specific areas and have publicly funded health insurance. As a Phase 2 trial, this research measures how well dupilumab works in an initial, smaller group, offering participants a chance to contribute to important findings.

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.

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

Research shows that dupilumab is generally safe for children and adults with moderate-to-severe asthma. For children aged 6-11, common side effects include injection site reactions, throat pain, and eye issues like redness and itching. These side effects are usually mild and manageable.

In studies with older children and adults (aged 12 and up), dupilumab significantly reduced severe asthma attacks. Some participants reported mild side effects like headaches and joint pain. The FDA has already approved dupilumab for treating asthma in older children and adults, indicating a good understanding of its safety.

Overall, research supports the treatment's safety in humans, but individual experiences can vary. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for asthma?

Dupilumab is unique because it targets a specific protein called IL-4 receptor alpha, which plays a key role in the inflammatory response that leads to asthma attacks. Unlike standard treatments for asthma, which often involve inhaled corticosteroids or bronchodilators that manage symptoms, Dupilumab works by reducing inflammation at its source. This targeted action not only helps in preventing asthma attacks but also has the potential to improve overall lung function in children. Researchers are excited about Dupilumab because it offers a new way to manage asthma by addressing the underlying causes rather than just alleviating symptoms.

What evidence suggests that dupilumab might be an effective treatment for asthma?

Research has shown that dupilumab, which participants in this trial may receive, helps prevent severe asthma attacks. In studies, patients using dupilumab experienced fewer severe asthma flare-ups compared to those taking a placebo (a harmless injection with no active medicine). Specifically, 75% of patients achieved better asthma control with dupilumab. It also improves lung function, measured by how well one can breathe out in one second. The treatment remains effective for up to 36 months. These findings suggest that dupilumab could be a promising option for children with asthma who often have flare-ups. ^{4 6 7 8 9}

Who Is on the Research Team?

Daniel J. Jackson, M.D.

Principal Investigator

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

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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 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]

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]

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, 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]

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 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 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]

Citations

1. dupixenthcp.com dupixenthcp.com/asthma/efficacy

Efficacy and Safety Overview 75% of patients had improved ACQ-5 (asthma control) scores with DUPIXENT 200 mg Q2W + SOC vs 67% with placebo + SOC (OR: 1.46 [95% CI: 0.90, 2.35]) (baseline ...

2. dupixent.com dupixent.com/asthma/

DUPIXENT® (dupilumab) in Moderate-to-Severe Asthma DUPIXENT helps prevent severe asthma attacks (exacerbations) and can improve your breathing. DUPIXENT may also help reduce the amount of oral corticosteroids ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9574251/

Dupilumab efficacy and safety in patients with moderate to ... Dupilumab significantly improved the absolute (dose-independent) FEV1 (L) at week 24 among 2,144 patients in the treatment group compared with ...

4. jaci-global.org jaci-global.org/article/S2772-8293(25)00134-1/fulltext

Long-term outcomes of dupilumab therapy in severe asthma Conclusions. Dupilumab represents an effective long-term treatment option for patients with severe asthma, with sustained treatment effects up to 36 months.

5. nejm.org nejm.org/doi/full/10.1056/NEJMoa1804092

Dupilumab Efficacy and Safety in Moderate-to-Severe ... In this trial, patients who received dupilumab had significantly lower rates of severe asthma exacerbation than those who received placebo, as well as better ...

6. dupixenthcp.com dupixenthcp.com/asthma/efficacy/children-safety-data

safety data in children (6-11 years) Find the safety profile, including common side effects, of DUPIXENT® (dupilumab) in pediatric patients aged 6-11 years with moderate-to-severe asthma ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT02277769

NCT02277769 | Study of Dupilumab (REGN668/ ... This is a randomized, double-blind, placebo-controlled, parallel-group study to confirm the efficacy and safety of Dupilumab monotherapy in adults with moderate ...

8. nejm.org nejm.org/doi/full/10.1056/NEJMoa1304048

Dupilumab in Persistent Asthma with Elevated Eosinophil ... Moderate-to-severe asthma remains poorly treated. We evaluated the efficacy and safety of dupilumab (SAR231893/REGN668), a fully human ...

9. dupixenthcp.com dupixenthcp.com/asthma/efficacy/safety-data

Safety Data For Asthma | DUPIXENT® (dupilumab) Explore safety data across clinical trials in patients aged 12+ with moderate-to-severe asthma characterized by an eosinophilic phenotype or with oral ...