Prevention of Asthma Exacerbations Using Dupilumab in Urban Children and Adolescents

NCT05347771 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: DAIT CAUSE-01
• Study Type: interventional
• Target: 240
• Locations: 1 country
• Sites: 8

Brief Summary

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.

Conditions

Asthma

Keywords

asthma; CAUSE; PANDA; T2; dupilumab

Outcome Measures

Primary Outcomes (1)

• Number of asthma exacerbations during the 12-month treatment period

  Asthma exacerbation defined as a prescription of a course of systemic steroids by a clinician or initiation of a course of systemic steroids by a participant to prevent a serious asthma outcome. If a participant initiates and completes a course of systemic steroids without clinician involvement, this course will be counted only if it meets the following dosage: the course for prednisone, prednisolone, or methylprednisolone will be at least 20 mg daily dose for 3 of 5 consecutive days. The course for dexamethasone will be at least a 10 mg single daily dose. If a corticosteroid burst for the treatment of an asthma exacerbation is prescribed by a non-CAUSE clinician, it will be counted regardless of dose.

  Week 4 (Treatment initiation) - Week 54 (Completion of treatment)

Secondary Outcomes (8)

• Pulmonary Function Measured by Spirometry: Forced Expiratory Volume in 1 Second (FEV1) % Predicted

  Week 4 to Week 68

• Days with symptoms, nights with symptoms, and day and night albuterol use.

  Week 4 to Week 68

• Asthma control measured by the Asthma Control Questionnaire-5

  Week 4 to Week 60

• Time to first asthma exacerbation

  Week 4 to Week 68

• Quality of life as measured by the PROMIS Asthma Impact Short Forms (Pediatric or Parent Proxy).

  Week 4 to Week 68

Study Arms (2)

Dupilumab

EXPERIMENTAL

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.

Drug: Dupilumab

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

Dupilumab DRUG

Dupilumab is a recombinant DNA-derived humanized IgG4ĸ monoclonal antibody that selectively binds to anti IL-4R monoclonal antibody (mAb).

Also known as: Dupixent, IL4Ra mAb

Dupilumab

Placebo DRUG

The composition of the placebo for dupilumab is the same as the active study drug without the dupilumab.

Also known as: Placebo for Dupilumab

Placebo

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant and/or parent guardian must be able to understand and provide informed consent and age-appropriate assent
• Are male and female aged 6-17 years at Visit 0
• Participant has a diagnosis of asthma made \> 1 year prior to recruitment. Participants who received an asthma diagnosis by a clinician ≤1 year prior to recruitment must report that their respiratory symptoms were present for more than 1 year prior to recruitment.
• Participant has had at least two asthma exacerbations in the prior year (defined as a requirement for systemic corticosteroids and/or hospitalization).
• At Visit 0 (screening), participant must have the following requirement for asthma controller medication:
• Participants aged 6 to 11 years: treatments with at least fluticasone 250 mcg dry powder inhaler (DPI) one puff twice daily or its equivalent.
• Participants aged 12 years and older, treatment with at least fluticasone 250 mcg plus long-acting beta agonist (LABA) DPI one puff twice daily or its equivalent.
• Have peripheral blood eosinophils ≥150 cells/mcl and/or FeNO ≥ 20ppb obtained at Visit 0 or via another CAUSE protocol within 6 months.
• Are able to perform acceptable and repeatable spirometry per American Thoracic Society (ATS) criteria prior to randomization.
• Have documentation of current medical insurance with prescription coverage at Visit 0.

You may not qualify if:

• Parent or guardian is not able or willing to give written informed consent or comply with study protocol.
• Have concurrent medical problems that would require systemic corticosteroids or other immunomodulators during the study.
• Are currently receiving immunotherapy.
• Are currently receiving treatment with a biologic therapy or have received a biologic therapy within 3 months prior to randomization.
• Are currently requiring greater than fluticasone 500 mcg bid plus long-acting beta agonist (LABA) one puff twice daily or its equivalent plus Long-acting muscarinic antagonists (LAMA) and/or individuals using oral corticosteroids daily or every other day for more than 14 days at the time of Visit 0.
• Are currently pregnant or lactating, or plan to become pregnant during the time of study participation. Participants of child-bearing potential (post-menarche) must be abstinent or use a medically acceptable birth control method throughout the study (i.e., oral subcutaneous, mechanical, or surgical contraception). Males who are sexually active must agree to use an acceptable method of birth control (i.e., barrier methods with vaginal spermicide) or have a partner practicing an approved birth control method.
• Have a known, pre-existing clinically important lung condition other than asthma.
• Have a current malignancy or previous history of cancer in remission for less than 12 months prior to randomization.
• Is a current smoker or is currently using any electronic cigarette or vaping device (e.g. e-cigarette, e- cig, mod, vape pen, JUUL, e-cigar, e-hookah, e-pipe, vape pods).
• Have a known immunodeficiency disease.
• Have a known, active pre-existing parasitic infection or are undergoing treatment for a parasitic infection. Once the participant has been successfully treated, the participant may be reevaluated.
• Use of investigational drugs within 4 weeks of randomization
• Have past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the site investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.
• Will not allow the study clinician, an asthma specialist, to manage their disease for the duration of the study or who is not willing to change their asthma medications to follow the Protocol CAUSE- 01 PANDA.
• Have a known history of allergic reaction to dupilumab.

Sponsors & Collaborators

• National Institute of Allergy and Infectious Diseases (NIAID): lead
• Childhood Asthma in Urban Settings (CAUSE): collaborator
• Regeneron Pharmaceuticals: collaborator
• Rho Federal Systems Division, Inc.: collaborator

Study Sites (8)

Children's Hospital Colorado: Allergy Program

Aurora, Colorado, 80045, United States

Location

Children's National Medical Center: Children's Research Institute

Washington D.C., District of Columbia, 20010, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago: Division of Allergy and Immunology

Chicago, Illinois, 60611, United States

Location

Boston Children's Hospital: Department of Immunology

Boston, Massachusetts, 02215, United States

Location

Washington University at St. Louis

St Louis, Missouri, 63110, United States

Location

Icahn School of Medicine at Mount Sinai: Division of Clinical Immunology, Immunology Institute

New York, New York, 10029, United States

Location

Columbia University Medical Center: Division of Pediatric Pulmonology

New York, New York, 10032, United States

Location

Cincinnati Children's Hospital Medical Center: Asthma Center

Cincinnati, Ohio, 45229, United States

Location

Related Links

• National Institute of Allergy and Infectious Diseases (NIAID)
• Division of Allergy, Immunology, and Transplantation (DAIT)

MeSH Terms

Conditions

Asthma

Interventions

dupilumab

Condition Hierarchy (Ancestors)

Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Respiratory Hypersensitivity; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Study Officials

• Daniel J. Jackson, M.D.

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

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 20, 2022
• First Posted: April 26, 2022
• Study Start: May 4, 2022
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): March 15, 2027
• Last Updated: January 20, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Time Frame: Post database lock
• Access Criteria: Open Access

Locations

US (8)