A Controlled Clinical Study of Dupilumab in Patients With Bilateral Nasal Polyps

NCT ID: NCT02912468

Last Updated: 2019-07-25

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

276 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-12-05

Study Completion Date

2018-07-05

Brief Summary

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Primary Objective:

To evaluate the efficacy of dupilumab 300 milligram (mg) every 2 weeks (q2w) compared to placebo on a background of mometasone furoate nasal spray (MFNS) in reducing nasal congestion/obstruction (NC) severity and endoscopic nasal polyp score (NPS) in participants with bilateral nasal polyposis (NP). In addition for Japan participants, reduction in computed tomography (CT) scan opacification of the sinuses was a coprimary objective.

Secondary Objectives:

* To evaluate the efficacy of dupilumab in improving total symptoms score (TSS).
* To evaluate the efficacy of dupilumab in improving sense of smell.
* To evaluate the efficacy of dupilumab in reducing CT scan opacification of the sinuses (primary objective for Japan).
* To evaluate ability of dupilumab in reducing proportion of participants requiring treatment with systemic corticosteroids or NP surgery.
* To evaluate the effect of dupilumab on participant reported outcomes and health related quality of life outcome by sinonasal outcome test-22 (SNOT-22).
* To evaluate the effect of dupilumab in the subgroups of participants with prior surgery and co-morbid asthma (including non-steroid antiinflammatory drug \[NSAID\] exacerbated respiratory disease \[ERD\]).
* To evaluate residual effect in follow up.
* To evaluate the safety of dupilumab in participants with bilateral NP.
* To evaluate functional dupilumab concentrations (systemic exposure) and incidence of treatment-emergent anti-drug antibodies.

Detailed Description

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The total study duration per participant was expected to be up to 52 weeks that consisted of a 4-weeks run-in period, 24-weeks treatment period, and a 24-weeks post treatment period.

Conditions

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Chronic Rhinosinusitis Phenotype With Nasal Polyps (CRSwNP)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators
Dupilumab and placebo were provided in matching 2 milliliter (mL) pre-filled syringes. To protect the blind, each treatment kit was prepared such that the dupilumab dose and its corresponding placebo volume were identical and indistinguishable and were labelled with a treatment kit number.

Study Groups

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Placebo

Placebo (for dupilumab), 1 subcutaneous (SC) injection every 2 weeks (q2w) from Day 1 of Week 0 up to Week 24 added to background therapy of intranasal mometasone furoate nasal spray (MFNS) at stable dose.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Pharmaceutical form: Solution

Route of administration: Subcutaneous

Mometasone furoate 50 micrograms

Intervention Type DRUG

Pharmaceutical form: Suspension (Nasal spray)

Route of administration: Intranasal

Dupilumab 300 mg

Dupilumab 300 mg SC injection q2w from Day 1 of Week 0 up to Week 24 added to background therapy of intranasal MFNS at stable dose.

Group Type EXPERIMENTAL

Dupilumab SAR231893 (REGN668)

Intervention Type DRUG

Pharmaceutical form: Solution

Route of administration: Subcutaneous

Mometasone furoate 50 micrograms

Intervention Type DRUG

Pharmaceutical form: Suspension (Nasal spray)

Route of administration: Intranasal

Interventions

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Dupilumab SAR231893 (REGN668)

Pharmaceutical form: Solution

Route of administration: Subcutaneous

Intervention Type DRUG

Placebo

Pharmaceutical form: Solution

Route of administration: Subcutaneous

Intervention Type DRUG

Mometasone furoate 50 micrograms

Pharmaceutical form: Suspension (Nasal spray)

Route of administration: Intranasal

Intervention Type DRUG

Other Intervention Names

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NASONEX®

Eligibility Criteria

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Inclusion Criteria

* Participants with bilateral sinonasal polyposis that despite prior treatment with systemic corticosteroids (SCS) anytime within the past 2 years; and/or had a medical contraindication / intolerance to SCS; and/or had prior surgery for NP at the screening visit, had:
* An endoscopic bilateral NPS of at least 5 out of a maximum score of 8 (with a minimum score of 2 in each nasal cavity).
* Ongoing symptoms (for at least 8 weeks prior to Visit \[V\] 1) of nasal congestion/blockage/obstruction with moderate or severe symptom severity (score 2 or 3) at V1 and a weekly average severity of greater than 1 at the time of randomization (V2), and another symptom such as loss of smell, rhinorrhea (anterior/posterior).
* Signed written informed consent.

Exclusion Criteria

* Participants \<18 years of age.
* Participants who were previously treated in dupilumab studies.
* Participants who had taken:
* Biologic therapy/systemic immunosuppressant to treat inflammatory disease or autoimmune disease (eg, rheumatoid arthritis, inflammatory bowel disease, primary biliary cirrhosis, systemic lupus erythematosus, multiple sclerosis, etc.) within 2 months before V1 or 5 half-lives, whichever was longer.
* Any experimental monoclonal antibody (mAB) within 5 half-lives or within 6 months before V1 if the half-life was unknown.
* Anti-immunoglobulin E (IgE) therapy (omalizumab) within 130 days prior to V1.
* Participants who received leukotriene antagonists/modifiers at V1 unless they were on a continuous treatment for at least 30 days prior to V1.
* Initiated allergen immunotherapy within 3 months prior to V1 or planned to begin therapy or changed its dose during the run-in period or the randomized treatment period.
* Participants who undergone any intranasal and/or sinus surgery (including polypectomy) within 6 months prior to V1.
* Participants who had a sinonasal or sinus surgery changing the lateral wall structure of the nose making impossible the evaluation of NPS.
* Participants with conditions/concomitant diseases making them nonevaluable at V1 or for the primary efficacy endpoint such as:
* Antrochoanal polyps;
* Nasal septal deviation that would occlude at least one nostril;
* Acute sinusitis, nasal infection or upper respiratory infection;
* Ongoing rhinitis medicamentosa;
* Allergic granulomatous angiitis (Churg-Strauss syndrome), granulomatosis with polyangiitis (Wegener's granulomatosis), Young's syndrome, Kartagener's syndrome or other dyskinetic ciliary syndromes, concomitant cystic fibrosis;
* Radiologic suspicion, or confirmed invasive or expansive fungal rhinosinusitis.
* Participants with nasal cavity malignant tumor and benign tumors (eg, papilloma, blood boil, etc).
* Participants with forced expiratory volume in 1 second (FEV1) 50% or less (of predicted normal).
* Participants who received concomitant treatment prohibited in the study.
* Treatment with a live (attenuated) vaccine within 12 weeks before the baseline visit.
* History of human immunodeficiency virus (HIV) infection or positive HIV serology at screening.
* Positive with hepatitis B surface antigen (HBsAg) or hepatitis C antibody at the screening visit.
* Active chronic or acute infection requiring systemic treatment within 2 weeks before the baseline visit.
* Known or suspected history of immunosuppression.
* Pregnant or breastfeeding women, or women planned to become pregnant or breastfeed during the study.
* Women unwilling to use adequate birth control, if of reproductive potential and sexually active.

The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Regeneron Pharmaceuticals

INDUSTRY

Sponsor Role collaborator

Sanofi

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

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Investigational Site Number 8400009

Long Beach, California, United States

Site Status

Investigational Site Number 8400004

Orange, California, United States

Site Status

Investigational Site Number 8400014

San Diego, California, United States

Site Status

Investigational Site Number 8400002

Stockton, California, United States

Site Status

Investigational Site Number 8400016

Centennial, Colorado, United States

Site Status

Investigational Site Number 8400013

Tampa, Florida, United States

Site Status

Investigational Site Number 8400022

West Des Moines, Iowa, United States

Site Status

Investigational Site Number 8400007

Boston, Massachusetts, United States

Site Status

Investigational Site Number 8400005

Rochester, Minnesota, United States

Site Status

Investigational Site Number 8400021

St Louis, Missouri, United States

Site Status

Investigational Site Number 8400008

Winston-Salem, North Carolina, United States

Site Status

Investigational Site Number 8400019

Tulsa, Oklahoma, United States

Site Status

Investigational Site Number 8400020

Medford, Oregon, United States

Site Status

Investigational Site Number 8400018

Charleston, South Carolina, United States

Site Status

Investigational Site Number 8400003

Nashville, Tennessee, United States

Site Status

Investigational Site Number 8400001

Dallas, Texas, United States

Site Status

Investigational Site Number 8400015

Norfolk, Virginia, United States

Site Status

Investigational Site Number 8400010

Milwaukee, Wisconsin, United States

Site Status

Investigational Site Number 1000003

Plovdiv, , Bulgaria

Site Status

Investigational Site Number 1000002

Sofia, , Bulgaria

Site Status

Investigational Site Number 1000001

Sofia, , Bulgaria

Site Status

Investigational Site Number 2030001

Hradec Králové, , Czechia

Site Status

Investigational Site Number 2030002

Pardubice, , Czechia

Site Status

Investigational Site Number 2030004

Prague, , Czechia

Site Status

Investigational Site Number 2500005

La Roche-sur-Yon, , France

Site Status

Investigational Site Number 2500007

Lille, , France

Site Status

Investigational Site Number 2500003

Lyon, , France

Site Status

Investigational Site Number 2500001

Montpellier, , France

Site Status

Investigational Site Number 2500006

Nantes, , France

Site Status

Investigational Site Number 2500002

Toulouse, , France

Site Status

Investigational Site Number 2500004

Vandœuvre-lès-Nancy, , France

Site Status

Investigational Site Number 2760001

Berlin, , Germany

Site Status

Investigational Site Number 2760003

München, , Germany

Site Status

Investigational Site Number 2760002

Münster, , Germany

Site Status

Investigational Site Number 3480006

Budapest, , Hungary

Site Status

Investigational Site Number 3480007

Budapest, , Hungary

Site Status

Investigational Site Number 3480004

Budapest, , Hungary

Site Status

Investigational Site Number 3480003

Budapest, , Hungary

Site Status

Investigational Site Number 3480005

Debrecen, , Hungary

Site Status

Investigational Site Number 3480002

Pécs, , Hungary

Site Status

Investigational Site Number 3480001

Szeged, , Hungary

Site Status

Investigational Site Number 3800007

Bologna, , Italy

Site Status

Investigational Site Number 3800002

Catania, , Italy

Site Status

Investigational Site Number 3800004

Milan, , Italy

Site Status

Investigational Site Number 3800006

Milan, , Italy

Site Status

Investigational Site Number 3800001

Pisa, , Italy

Site Status

Investigational Site Number 3800005

Rozzano, , Italy

Site Status

Investigational Site Number 3800003

Varese, , Italy

Site Status

Investigational Site Number 5280001

Amsterdam, , Netherlands

Site Status

Investigational Site Number 6160002

Katowice, , Poland

Site Status

Investigational Site Number 6160001

Lodz, , Poland

Site Status

Investigational Site Number 6160003

Warsaw, , Poland

Site Status

Investigational Site Number 6420007

Brasov, , Romania

Site Status

Investigational Site Number 6420013

Brasov, , Romania

Site Status

Investigational Site Number 6420003

Bucharest, , Romania

Site Status

Investigational Site Number 6420009

Cluj-Napoca, , Romania

Site Status

Investigational Site Number 6420008

Craiova, , Romania

Site Status

Investigational Site Number 6420010

Târgu Mureş, , Romania

Site Status

Investigational Site Number 6430004

Moscow, , Russia

Site Status

Investigational Site Number 6430003

Moscow, , Russia

Site Status

Investigational Site Number 6430002

Saint Petersburg, , Russia

Site Status

Investigational Site Number 6430001

Saint Petersburg, , Russia

Site Status

Investigational Site Number 6430007

Saint Petersburg, , Russia

Site Status

Investigational Site Number 6430006

Yaroslavl, , Russia

Site Status

Investigational Site Number 8040002

Ivano-Frankivsk, , Ukraine

Site Status

Investigational Site Number 8040004

Kharkiv, , Ukraine

Site Status

Investigational Site Number 8040005

Kyiv, , Ukraine

Site Status

Investigational Site Number 8040006

Kyiv, , Ukraine

Site Status

Investigational Site Number 8040001

Poltava, , Ukraine

Site Status

Investigational Site Number 8040008

Ternopil, , Ukraine

Site Status

Investigational Site Number 8260001

Bradford, , United Kingdom

Site Status

Investigational Site Number 8260002

Dundee, , United Kingdom

Site Status

Investigational Site Number 8260007

Great Yarmouth, , United Kingdom

Site Status

Investigational Site Number 8260006

London, , United Kingdom

Site Status

Investigational Site Number 8260004

Stockport, , United Kingdom

Site Status

Investigational Site Number 8260005

Wigan, , United Kingdom

Site Status

Countries

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United States Bulgaria Czechia France Germany Hungary Italy Netherlands Poland Romania Russia Ukraine United Kingdom

References

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Lane AP, Mullol J, Hopkins C, Fokkens WJ, Lee SE, Msihid J, Nash S, Sacks H, Borsos K, Kamat S, Rowe PJ, Deniz Y, Jacob-Nara JA. Dupilumab improves sense of smell and clinical outcomes in patients with severe chronic rhinosinusitis with nasal polyps with anosmia. Curr Med Res Opin. 2025 Jan;41(1):53-59. doi: 10.1080/03007995.2024.2434083. Epub 2024 Dec 14.

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Bachert C, Khan AH, Hopkins C, Han JK, Fokkens WJ, Mannent LP, Msihid J, Borsos K, Kamat S, Nash S, Sacks H, Rowe PJ, Deniz Y, Jacob-Nara JA. Mild and symptom-free months in patients with chronic rhinosinusitis with nasal polyps treated with dupilumab. Ann Allergy Asthma Immunol. 2025 Jan;134(1):61-69.e12. doi: 10.1016/j.anai.2024.09.015. Epub 2024 Sep 28.

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Hopkins C, Mullol J, Khan AH, Lee SE, Wagenmann M, Hellings P, Fokkens W, Msihid J, Nair R, Kamat S, Nash S, Radwan A, Jacob-Nara JA, Deniz Y, Rowe PJ. Impact of Dupilumab on Sinonasal Symptoms and Outcomes in Severe Chronic Rhinosinusitis With Nasal Polyps. Otolaryngol Head Neck Surg. 2024 Apr;170(4):1173-1182. doi: 10.1002/ohn.627. Epub 2023 Dec 29.

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Maspero JF, Bachert C, Martinez FJ, Hanania NA, Ortiz B, Patel N, Mannent LP, Praestgaard A, Pandit-Abid N, Siddiqui S, Hardin M. Clinical Efficacy among Patients with Chronic Rhinosinusitis with Nasal Polyps and Clinical Features of Obstructive Lung Disease: Post Hoc Analysis of the Phase III SINUS-24 and SINUS-52 Studies. J Asthma Allergy. 2023 Mar 31;16:333-342. doi: 10.2147/JAA.S357393. eCollection 2023.

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Peters AT, Soler ZM, Kern RC, Heffler E, Maspero JF, Crampette L, Fujieda S, Lane AP, Zhang H, Nash S, Khan AH, Siddiqui S, Jacob-Nara JA, Rowe P, Deniz Y. Improvement in patient-reported "taste" and association with smell in dupilumab-treated patients with severe chronic rhinosinusitis with nasal polyps from the SINUS-24 and SINUS-52 trials. Clin Exp Allergy. 2022 Sep;52(9):1105-1109. doi: 10.1111/cea.14194. Epub 2022 Jul 12. No abstract available.

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Wechsler ME, Klion AD, Paggiaro P, Nair P, Staumont-Salle D, Radwan A, Johnson RR, Kapoor U, Khokhar FA, Daizadeh N, Chen Z, Laws E, Ortiz B, Jacob-Nara JA, Mannent LP, Rowe PJ, Deniz Y. Effect of Dupilumab on Blood Eosinophil Counts in Patients With Asthma, Chronic Rhinosinusitis With Nasal Polyps, Atopic Dermatitis, or Eosinophilic Esophagitis. J Allergy Clin Immunol Pract. 2022 Oct;10(10):2695-2709. doi: 10.1016/j.jaip.2022.05.019. Epub 2022 May 28.

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PMID: 35636689 (View on PubMed)

Bachert C, Peters AT, Heffler E, Han JK, Olze H, Pfaar O, Chuang CC, Rout R, Attre R, Goga L, Jacob-Nara JA, Rowe PJ, Deniz Y, Chen Z, Kamat S, Siddiqui S. Responder analysis to demonstrate the effect of targeting type 2 inflammatory mechanisms with dupilumab across objective and patient-reported endpoints for patients with severe chronic rhinosinusitis with nasal polyps in the SINUS-24 and SINUS-52 studies. Clin Exp Allergy. 2022 Feb;52(2):244-249. doi: 10.1111/cea.14051. No abstract available.

Reference Type DERIVED
PMID: 35092110 (View on PubMed)

Lee SE, Hopkins C, Mullol J, Msihid J, Guillemin I, Amin N, Mannent LP, Li Y, Siddiqui S, Chuang CC, Kamat S, Khan AH. Dupilumab improves health related quality of life: Results from the phase 3 SINUS studies. Allergy. 2022 Jul;77(7):2211-2221. doi: 10.1111/all.15222. Epub 2022 Feb 1.

Reference Type DERIVED
PMID: 35034364 (View on PubMed)

Bachert C, Corren J, Lee SE, Zhang H, Harel S, Cunoosamy D, Khan AH, Jacob-Nara JA, Siddiqui S, Nash S, Rowe PJ, Deniz Y. Dupilumab efficacy and biomarkers in chronic rhinosinusitis with nasal polyps: Association between dupilumab treatment effect on nasal polyp score and biomarkers of type 2 inflammation in patients with chronic rhinosinusitis with nasal polyps in the phase 3 SINUS-24 and SINUS-52 trials. Int Forum Allergy Rhinol. 2022 Sep;12(9):1191-1195. doi: 10.1002/alr.22964. Epub 2022 Jan 31. No abstract available.

Reference Type DERIVED
PMID: 34970860 (View on PubMed)

Hellings PW, Peters AT, Chaker AM, Heffler E, Zhang H, Praestgaard A, Nash S, Khan AH, Siddiqui S, Jacob-Nara JA, Rowe PJ, Deniz Y. Rapid and sustained effects of dupilumab in severe chronic rhinosinusitis with nasal polyps. Int Forum Allergy Rhinol. 2022 Jul;12(7):958-962. doi: 10.1002/alr.22944. Epub 2022 Jan 23. No abstract available.

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PMID: 34911163 (View on PubMed)

Han JK, Bachert C, Lee SE, Hopkins C, Heffler E, Hellings PW, Peters AT, Kamat S, Whalley D, Qin S, Nelson L, Siddiqui S, Khan AH, Li Y, Mannent LP, Guillemin I, Chuang CC. Estimating Clinically Meaningful Change of Efficacy Outcomes in Inadequately Controlled Chronic Rhinosinusitis with Nasal Polyposis. Laryngoscope. 2022 Feb;132(2):265-271. doi: 10.1002/lary.29888. Epub 2021 Dec 1.

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PMID: 34850966 (View on PubMed)

Chuang CC, Guillemin I, Bachert C, Lee SE, Hellings PW, Fokkens WJ, Duverger N, Fan C, Daizadeh N, Amin N, Mannent LP, Khan AH, Kamat S. Dupilumab in CRSwNP: Responder Analysis Using Clinically Meaningful Efficacy Outcome Thresholds. Laryngoscope. 2022 Feb;132(2):259-264. doi: 10.1002/lary.29911. Epub 2021 Nov 24.

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Mullol J, Laidlaw TM, Bachert C, Mannent LP, Canonica GW, Han JK, Maspero JF, Picado C, Daizadeh N, Ortiz B, Li Y, Ruddy M, Laws E, Amin N. Efficacy and safety of dupilumab in patients with uncontrolled severe chronic rhinosinusitis with nasal polyps and a clinical diagnosis of NSAID-ERD: Results from two randomized placebo-controlled phase 3 trials. Allergy. 2022 Apr;77(4):1231-1244. doi: 10.1111/all.15067. Epub 2021 Oct 1.

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PMID: 34459002 (View on PubMed)

Khan AH, Reaney M, Guillemin I, Nelson L, Qin S, Kamat S, Mannent L, Amin N, Whalley D, Hopkins C. Development of Sinonasal Outcome Test (SNOT-22) Domains in Chronic Rhinosinusitis With Nasal Polyps. Laryngoscope. 2022 May;132(5):933-941. doi: 10.1002/lary.29766. Epub 2021 Aug 26.

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PMID: 34437720 (View on PubMed)

Chong LY, Piromchai P, Sharp S, Snidvongs K, Webster KE, Philpott C, Hopkins C, Burton MJ. Biologics for chronic rhinosinusitis. Cochrane Database Syst Rev. 2021 Mar 12;3(3):CD013513. doi: 10.1002/14651858.CD013513.pub3.

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PMID: 33710614 (View on PubMed)

Hopkins C, Wagenmann M, Bachert C, Desrosiers M, Han JK, Hellings PW, Lee SE, Msihid J, Radwan A, Rowe P, Amin N, Deniz Y, Ortiz B, Mannent LP, Rout R. Efficacy of dupilumab in patients with a history of prior sinus surgery for chronic rhinosinusitis with nasal polyps. Int Forum Allergy Rhinol. 2021 Jul;11(7):1087-1101. doi: 10.1002/alr.22780. Epub 2021 Feb 21.

Reference Type DERIVED
PMID: 33611847 (View on PubMed)

Peters AT, Han JK, Hellings P, Heffler E, Gevaert P, Bachert C, Xu Y, Chuang CC, Neupane B, Msihid J, Mannent LP, Guyot P, Kamat S. Indirect Treatment Comparison of Biologics in Chronic Rhinosinusitis with Nasal Polyps. J Allergy Clin Immunol Pract. 2021 Jun;9(6):2461-2471.e5. doi: 10.1016/j.jaip.2021.01.031. Epub 2021 Feb 4.

Reference Type DERIVED
PMID: 33548517 (View on PubMed)

Laidlaw TM, Bachert C, Amin N, Desrosiers M, Hellings PW, Mullol J, Maspero JF, Gevaert P, Zhang M, Mao X, Khan AH, Kamat S, Patel N, Graham NMH, Ruddy M, Staudinger H, Mannent LP. Dupilumab improves upper and lower airway disease control in chronic rhinosinusitis with nasal polyps and asthma. Ann Allergy Asthma Immunol. 2021 May;126(5):584-592.e1. doi: 10.1016/j.anai.2021.01.012. Epub 2021 Jan 16.

Reference Type DERIVED
PMID: 33465455 (View on PubMed)

Bachert C, Han JK, Desrosiers M, Hellings PW, Amin N, Lee SE, Mullol J, Greos LS, Bosso JV, Laidlaw TM, Cervin AU, Maspero JF, Hopkins C, Olze H, Canonica GW, Paggiaro P, Cho SH, Fokkens WJ, Fujieda S, Zhang M, Lu X, Fan C, Draikiwicz S, Kamat SA, Khan A, Pirozzi G, Patel N, Graham NMH, Ruddy M, Staudinger H, Weinreich D, Stahl N, Yancopoulos GD, Mannent LP. Efficacy and safety of dupilumab in patients with severe chronic rhinosinusitis with nasal polyps (LIBERTY NP SINUS-24 and LIBERTY NP SINUS-52): results from two multicentre, randomised, double-blind, placebo-controlled, parallel-group phase 3 trials. Lancet. 2019 Nov 2;394(10209):1638-1650. doi: 10.1016/S0140-6736(19)31881-1. Epub 2019 Sep 19.

Reference Type DERIVED
PMID: 31543428 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2015-003101-42

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1178-5390

Identifier Type: OTHER

Identifier Source: secondary_id

EFC14146

Identifier Type: -

Identifier Source: org_study_id

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