Investigating Dupilumab's Effect in Asthma by Genotype

NCT03694158 | Active Not Recruiting Phase 4 DMC FDA Drug

• 2 other identifiers: P00029072U01AI143514
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 6

Brief Summary

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.

Conditions

Asthma

Outcome Measures

Primary Outcomes (1)

• The rate of asthma exacerbations

  An exacerbation is an asthma attack for which a clinician prescribed a course of systemic steroids, whether or not the patient took the steroids.

  48 week treatment period

Secondary Outcomes (2)

• Change in pre-bronchodilator lung function

  average of week 4,12, 24,36 and 48 week

• Change in CASI score

  average of 4,12, 24, 36, and 48 week

Study Arms (2)

Treatment group

EXPERIMENTAL

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.

Drug: Dupilumab

Placebo group

PLACEBO COMPARATOR

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

Other: Placebo

Interventions

Dupilumab DRUG

anti-IL4 receptor antagonist

Also known as: Dupixent®

Treatment group

Placebo OTHER

Placebo for Dupilumab (packaged/administered the same as the active drug)

Placebo group

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Ages 12 years and older
• Ability to provide informed consent
• Ability to perform pulmonary function tests
• Female participants of childbearing potential must have a negative urine pregnancy test upon study entry
• Female participants with reproductive potential must agree to use FDA-approved methods of birth control for the duration of the study2
• Participant-reported physician or licensed medical practitioner diagnosis of asthma
• Treatment with medium to high dose ICS (400 mcg to maximum of 2000 mcg per day of fluticasone propionate or equivalent) for at least 3 months with a stable dose ≥1 month prior to screening OR used a biologic medication for asthma within the past 8 weeks
• History of asthma exacerbation in the past year
• An exacerbation is an asthma attack for which a clinician prescribed a course of systemic (oral, IV, IM) steroids whether or not the patient took the steroids OR An increase of \>50% of baseline inhaled corticosteroid dose for ≥3 days OR An unscheduled visit for acute asthma attack (licensed medical practitioner/nurse office, urgent care intervention, emergency department, or hospitalization)

You may not qualify if:

• Chronic lung disease other than asthma, which may impair lung function
• Current smoker or cessation of smoking ≤6 months prior to Visit 0 screening
• Current use of any electronic (e) "vaping" device (e.g., e-cigarette, e-cig, mod, vape pen, JUUL, e-cigar, e-hookah, e-pipe, vape pods) or cessation ≤ 6 months prior to screening
• Pregnant or breast feeding
• Any other condition or abnormality that, in the opinion of the Principal Investigator, would compromise the safety of the patient or quality of data
• Evidence that the participant or family may be unreliable or poorly adherent to their asthma treatment or study procedures
• Planning to relocate away from the clinical center area before study completion
• Currently participating in an investigational drug trial or participated in one within 30 days before screening
• Currently being treated with immunosuppressive/immunomodulatory or other investigational agents or biologics for conditions other than asthma, or used a biologic for a non-asthma indication within the past 6 months
• History of respiratory illness requiring antibiotics or systemic corticosteroids, including asthma exacerbations, within the past 4 weeks (evaluated at time of screening visit)
• History of alcohol or illicit substance abuse within 6 months of screening
• Neutropenia (\<1,000/mm3) or thrombocytopenia (\<100,000/mm3) or hemoglobin \< 100 g/L (10 g/dL) or blood eosinophils \> 1500/mm3 at screening
• Administration of a live vaccine within 4 weeks of screening
• Currently receiving allergen immunotherapy (food or aeroallergen) other than an established maintenance regimen implemented continuously for a minimum of 2 months. Individuals receiving aeroallergen immunotherapy must be willing to stay on it for the duration of the study.

Sponsors & Collaborators

• Boston Children's Hospital: lead
• National Institute of Allergy and Infectious Diseases (NIAID): collaborator
• Regeneron Pharmaceuticals: collaborator
• HealthBeacon Plc: collaborator
• Merck Sharp & Dohme LLC: collaborator
• Sanofi: collaborator

Study Sites (6)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Montefiore Einstein Clinical Research Center

The Bronx, New York, 10467, United States

Location

MetroHealth System

Cleveland, Ohio, 44109, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (2)

• Lai PS, Massoud AH, Xia M, Petty CR, Cunningham A, Chatila TA, Phipatanakul W. Gene-environment interaction between an IL4R variant and school endotoxin exposure contributes to asthma symptoms in inner-city children. J Allergy Clin Immunol. 2018 Feb;141(2):794-796.e3. doi: 10.1016/j.jaci.2017.08.023. Epub 2017 Sep 21. No abstract available.

  PMID: 28943468 BACKGROUND

• Massoud AH, Charbonnier LM, Lopez D, Pellegrini M, Phipatanakul W, Chatila TA. An asthma-associated IL4R variant exacerbates airway inflammation by promoting conversion of regulatory T cells to TH17-like cells. Nat Med. 2016 Sep;22(9):1013-22. doi: 10.1038/nm.4147. Epub 2016 Aug 1.

  PMID: 27479084 BACKGROUND

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

• Wanda Phipatanakul, MD, MS

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double blind, placebo controlled.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Pediatrics

Model Details: This is a genotype stratified, double-blind, randomized, placebo-controlled, parallel-group, phase IV clinical trial

Study Record Dates

• First Submitted: October 1, 2018
• First Posted: October 3, 2018
• Study Start: September 8, 2021
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: February 23, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)