AsthmaPhase 4IDEANCT03694158
Investigating Dupilumab's Effect in Asthma by Genotype
A Phase 4 study testing Dupilumab in people with Asthma, run by Boston Children's Hospital at 6 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Asthma
- What is being tested?
- Dupilumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 150 participants
- Where
- 6 sitesUnited States
- Study dates
- 8 Sep 2021to Mar 2027
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Show all 9Show fewer
- 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)
Who cannot join
- 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
Show all 14Show fewer
- 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.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
Brigham and Women's Hospital
Boston, Massachusetts, 02115
MetroHealth System
Cleveland, Ohio, 44109
Henry Ford Health System
Detroit, Michigan, 48202
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Montefiore Einstein Clinical Research Center
The Bronx, New York, 10467
About this study
Official title: Effect of IL-4RαR576 Polymorphism on Response to Dupilumab in Asthma, a Genotype-stratified, Randomized, Placebo- Controlled Trial
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.
Read the detailed description
This is a double-blind, randomized, placebo-controlled parallel-group phase 4 clinical trial.
Patients will be genotyped and categorized as those with: 1) the wild type allele (Q576/Q576), 2) heterozygous allele (Q576/R576), or 3) homozygous mutant allele (R576/R576); the genotype associated with more severe disease.
After a run-in period of 2-12 weeks to determine asthma control, subjects who fulfill all inclusion/exclusion criteria will be randomized to receive either subcutaneous Dupilumab or placebo (1:1 randomization allocation ratio).
This study addresses fundamental mechanisms by which the IL-4Rα-R576 variant drives the TH2/TH17 disease endotype and the influence of this variant on response to Dupilumab therapy. It brings together individuals with deep clinical and scientific expertise in allergic diseases, including epidemiology, genetics, inflammation, and tolerance mechanisms to investigate, in a coordinated strategy, the hypothesis that the IL-4Rα-R576 variant drives TH2/TH17 cell inflammation by subverting allergen-specific iTreg cells into TH17 cells. Asthmatics bearing this endotype will be particularly likely to favorably respond to Dupilumab therapy by virtue of its prevention of iTreg cell reprogramming into TH17-like cells, potentially leading to their long-term stability and potential for sustained immune tolerance.
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DupilumabDrug
Also called Dupixent®
anti-IL4 receptor antagonist
PlaceboOther
Placebo for Dupilumab (packaged/administered the same as the active drug)
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Dupilumab |
| Placebo group | Placebo comparator | Placebo (preparation, administration, packaging, and labeling all equivalent to the treatment) administered subcutaneously every two weeks. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 48 week treatment period
Secondary outcomes (2)
Change in pre-bronchodilator lung function
the change in pre-bronchodilator FEV1% predicted from baseline
Measured: average of week 4,12, 24,36 and 48 week
Change in CASI score
The change in CASI score from baseline
Measured: average of 4,12, 24, 36, and 48 week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2018
- Study start8 Sep 2021
- Primary completionJan 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for AsthmaStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This is a genotype stratified, double-blind, randomized, placebo-controlled, parallel-group, phase IV clinical trial
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Allergy and Infectious Diseases (NIAID), Regeneron Pharmaceuticals, HealthBeacon Plc, Merck Sharp & Dohme LLC, Sanofi
- Responsible party
- Principal investigator, Wanda Phipatanakul, Harvard Medical School (HMS and HSDM)
- Investigators
- Wanda Phipatanakul, MD, MS, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03694158
- Sponsor's protocol ID
- P00029072
- Other IDs
- U01AI143514
- Acronym
- IDEA
- Status last verified
- Feb 2026
- First submitted
- 1 Oct 2018
- Last updated
- 23 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 28943468Massoud 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.
PubMed 27479084Phipatanakul W, Israel E, Mauger DT, Kunselman SJ, Becker PM, Gagalis L, Dabek RL, Greenwood J, Kim H, Zoratti E, Kaelber DC, Ayache M, Jariwala S, Banerjee A, Oppenheimer J, Gaffin JM, Lai PS, Banzon TM, Thorne PS, Oettgen HC, Lee P, Lam M, Chatila T. Effect of IL-4RalphaR576 polymorphism on response to dupilumab in asthma, a genotype-stratified, randomized, placebo- controlled trial (IDEA Trial): Design, Rationale, Methods, Strategies, and Lessons Learned. J Asthma. 2026 Sep 15:1-24. doi: 10.1080/02770903.2026.2734972. Online ahead of print.
PubMed 42742633
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of IL-4RαR576 Polymorphism on Response to Dupilumab in Asthma, a Genotype-stratified, Randomized, Placebo- Controlled Trial
- ClinicalTrials.gov ID
- NCT03694158
- Acronym / sponsor ID
- IDEA / P00029072
- Conditions
- Asthma
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- National Institute of Allergy and Infectious Diseases (NIAID), Regeneron Pharmaceuticals, HealthBeacon Plc, Merck Sharp & Dohme LLC, Sanofi
- Study start
- 8 Sep 2021 (actual)
- Primary completion
- Jan 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 3 Oct 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a double-blind, randomized, placebo-controlled parallel-group phase 4 clinical trial.
Patients will be genotyped and categorized as those with: 1) the wild type allele (Q576/Q576), 2) heterozygous allele (Q576/R576), or 3) homozygous mutant allele (R576/R576); the genotype associated with more severe disease.
After a run-in period of 2-12 weeks to determine asthma control, subjects who fulfill all inclusion/exclusion criteria will be randomized to receive either subcutaneous Dupilumab or placebo (1:1 randomization allocation ratio).
This study addresses fundamental mechanisms by which the IL-4Rα-R576 variant drives the TH2/TH17 disease endotype and the influence of this variant on response to Dupilumab therapy. It brings together individuals with deep clinical and scientific expertise in allergic diseases, including epidemiology, genetics, inflammation, and tolerance mechanisms to investigate, in a coordinated strategy, the hypothesis that the IL-4Rα-R576 variant drives TH2/TH17 cell inflammation by subverting allergen-specific iTreg cells into TH17 cells. Asthmatics bearing this endotype will be particularly likely to favorably respond to Dupilumab therapy by virtue of its prevention of iTreg cell reprogramming into TH17-like cells, potentially leading to their long-term stability and potential for sustained immune tolerance.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- This is a genotype stratified, double-blind, randomized, placebo-controlled, parallel-group, phase IV clinical trial
| Arm | Type | Receives |
|---|---|---|
| Treatment group | Experimental | Drug: DupilumabDupilumab (Dupixent®) administered subcutaneously every two weeks. An initial dose of 600 mg (two 300 mg injections) followed by 300 mg given every other week. |
| Placebo group | Placebo comparator | Other: PlaceboPlacebo (preparation, administration, packaging, and labeling all equivalent to the treatment) administered subcutaneously every two weeks. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DupilumabAlso: Dupixent®anti-IL4 receptor antagonist | Drug | Treatment group |
| PlaceboPlacebo for Dupilumab (packaged/administered the same as the active drug) | Other | Placebo group |
Primary outcome
The rate of asthma exacerbations
Time frame: 48 week treatment period
An exacerbation is an asthma attack for which a clinician prescribed a course of systemic steroids, whether or not the patient took the steroids.
2 secondary outcomes
Change in pre-bronchodilator lung function
Time frame: average of week 4,12, 24,36 and 48 week
Change in CASI score
Time frame: average of 4,12, 24, 36, and 48 week
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 2026.
- Background: PubMed 28943468Lai 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.
- Background: PubMed 27479084Massoud 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.
- Derived from this study: PubMed 42742633Phipatanakul W, Israel E, Mauger DT, Kunselman SJ, Becker PM, Gagalis L, Dabek RL, Greenwood J, Kim H, Zoratti E, Kaelber DC, Ayache M, Jariwala S, Banerjee A, Oppenheimer J, Gaffin JM, Lai PS, Banzon TM, Thorne PS, Oettgen HC, Lee P, Lam M, Chatila T. Effect of IL-4RalphaR576 polymorphism on response to dupilumab in asthma, a genotype-stratified, randomized, placebo- controlled trial (IDEA Trial): Design, Rationale, Methods, Strategies, and Lessons Learned. J Asthma. 2026 Sep 15:1-24. doi: 10.1080/02770903.2026.2734972. Online ahead of print.
- Individual participant dataSharing planned: No
Related trials for Asthma
RecruitingPhase 1
Pilot Study to Identify the Mediators and Inflammatory Cell Surface Receptors Involved in Allergic Airway Inflammation
Asthma
RecruitingPhase 1
A Study to Compare the Relative Potency of Salbutamol Administered Via Metered Dose Inhalers (MDI) Containing Propellants HFA-152a to HFA-134a in Mild Asthmatics Aged 18 to 65 Inclusive
Asthma, Mild Asthma
RecruitingPhase 3
A Study Assessing the Effect of Dupilumab on Inducing Clinical Remission in Asthma
Asthma Control
Active, not recruitingPhase 2/3
Local Antibiotic Delivery for Community Acquired Pneumonia
Community Acquired Pneumonia (CAP)
RecruitingPhase 2
A Study to Learn About the Study Medicine Called PF-07275315 in People With Moderate-To-Severe Asthma
Asthma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wanda Phipatanakul, MD, MS principal investigator
Boston Children's Hospital
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Dupilumab in people with Asthma, run by Boston Children's Hospital at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 23 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The rate of asthma exacerbations (time frame: 48 week treatment period). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03694158, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Feb 2026.