NCT05331755

Brief Summary

Rationale: Dupilumab has proven to be effective and safe in several large randomized controlled trials. However, study populations in RCT's represent only a small proportion of severe asthma patients treated in real life circumstances. Therefore, real-life studies provide data complementary to RCT's derived from a more diverse and heterogeneous group of patients. This study is unique in that it represents a large population of European patients that are not represented in the Global Registry. Objective: To collect and analyse efficacy and safety data in severe asthma patients treated with dupilumab in a real-life setting. Study design: Single center, retrospective cohort analysis of severe asthma patients treated with dupilumab, as decided by the treating physician. Setting: Severe Asthma Expert Center Franciscus Gasthuis \& Vlietland, Rotterdam Study population: All patients that started with dupilumab from jan 2019 (early access program) until June 2020 are eligible for the study. They should be 18 years or older, with severe T2 high asthma and treated with at least one dose of dupilumab. Main study parameters/endpoints:

  • Primary endpoint: To assess changes in annual exacerbation rate between baseline and after 12 months treatment in severe asthma patients treated with dupilumab.
  • Secondary endpoints: To collect data on ACQ, OCS use, FEV1, treatment response rate, influence of co-morbidities and safety. Methods: Exacerbation history and patient characterization will be done at baseline. We aim to include patients in the analysis that had at least one administration of dupilumab (intention-to-treat). Treatment response evaluation is done at 12 months. Final evaluation of efficacy and safety is done at 12 months of treatment. We aim to enroll at least 120 patients in the study. Data collection will be done in Castor Electronic Data Capture. Of the 120 patients scheduled for inclusion, all have already started treatment and data will be collected retrospectively at the predefined time points. Patient data will be handled confidentially. Nature and extent of the burden and risks associated with participation, benefit and group relatedness: Since this is a retrospective cohort analysis there are no extra visits, interventions or procedures needed. Therefore, there is no increased burden or risk for the patients involved.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
120

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Apr 2021

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2021

Completed
1 year until next milestone

First Submitted

Initial submission to the registry

April 2, 2022

Completed
16 days until next milestone

First Posted

Study publicly available on registry

April 18, 2022

Completed
7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 30, 2022

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2022

Completed
Last Updated

September 21, 2023

Status Verified

September 1, 2023

Enrollment Period

1.6 years

First QC Date

April 2, 2022

Last Update Submit

September 19, 2023

Conditions

Severe Asthma

Outcome Measures

Primary Outcomes (1)

  • Annual exacerbation rate

    To assess changes in annual exacerbation rate prior to and after 12 months treatment in severe asthma patients treated with dupilumab.

    12 months before and after start medication

Secondary Outcomes (2)

  • Change in lung function

    12 months before and after start medication

  • Change in asthma control

    12 months before and after start medication

Other Outcomes (8)

  • % of patients with atopy

    12 months before and after start medication

  • % patients referred from other hospitals

    12 months before and after start medication

  • Responder rate and average treatment time with dupilumab

    12 months before and after start medication

  • +5 more other outcomes

Study Arms (1)

Severe T2 high asthma

Included patients will have severe type 2 high asthma, are eligible for dupilumab conform EU label, minimally treated with high dose ICS and LABA and have: * blood eosinophils 150 - 1500/mm2 OR * FeNO \>20 ppb OR * clinical significant inhalation allergy (+ sIgE positive) OR * OCS dependency

Drug: Dupilumab

Interventions

DupilumabDRUG

start medication in cohort

Also known as: Dupixent
Severe T2 high asthma

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Included patients will have severe type 2 high asthma, are eligible for dupilumab conform EU label, minimally treated with high dose ICS and LABA and have: * blood eosinophils 150 - 1500/mm2 OR * FeNO \>20 ppb OR * clinical significant inhalation allergy (+ sIgE positive) OR * OCS dependency

You may qualify if:

  • All patients that started with dupilumab from Jan 2019 (early access program) until June 2020 are eligible for the study. They should be 18 years or older, with severe T2 high asthma and treated with at least one dose of dupilumab. All included patients should provide written informed consent.

You may not qualify if:

  • Patients with COPD (Chronic Obstructive Pulmonary Disease) or EGPA (Eosinophilic Granulomatosis with PolyAngiitis) will be excluded.
  • Also, patients that had dupilumab prescribed for other reasons (nasal polyposis or atopic dermatitis) will be excluded as well.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Franciscus Gasthuis & Vlietland

Rotterdam, 3045PM, Netherlands

Location

Related Publications (8)

  • 4. Update asthma guidelines 2020 (Global Initiative for Asthma): www.ginasthma.org.

    BACKGROUND

  • Dupin C, Belhadi D, Guilleminault L, Gamez AS, Berger P, De Blay F, Bonniaud P, Leroyer C, Mahay G, Girodet PO, Raherison C, Fry S, Le Bourdelles G, Proust A, Rosencher L, Garcia G, Bourdin A, Chenivesse C, Didier A, Couffignal C, Taille C. Effectiveness and safety of dupilumab for the treatment of severe asthma in a real-life French multi-centre adult cohort. Clin Exp Allergy. 2020 Jul;50(7):789-798. doi: 10.1111/cea.13614. Epub 2020 May 29.

    PMID: 32469092BACKGROUND

  • Hekking PW, Wener RR, Amelink M, Zwinderman AH, Bouvy ML, Bel EH. The prevalence of severe refractory asthma. J Allergy Clin Immunol. 2015 Apr;135(4):896-902. doi: 10.1016/j.jaci.2014.08.042. Epub 2014 Oct 16.

    PMID: 25441637RESULT

  • Rabe KF, Nair P, Brusselle G, Maspero JF, Castro M, Sher L, Zhu H, Hamilton JD, Swanson BN, Khan A, Chao J, Staudinger H, Pirozzi G, Antoni C, Amin N, Ruddy M, Akinlade B, Graham NMH, Stahl N, Yancopoulos GD, Teper A. Efficacy and Safety of Dupilumab in Glucocorticoid-Dependent Severe Asthma. N Engl J Med. 2018 Jun 28;378(26):2475-2485. doi: 10.1056/NEJMoa1804093. Epub 2018 May 21.

    PMID: 29782224RESULT

  • Castro M, Corren J, Pavord ID, Maspero J, Wenzel S, Rabe KF, Busse WW, Ford L, Sher L, FitzGerald JM, Katelaris C, Tohda Y, Zhang B, Staudinger H, Pirozzi G, Amin N, Ruddy M, Akinlade B, Khan A, Chao J, Martincova R, Graham NMH, Hamilton JD, Swanson BN, Stahl N, Yancopoulos GD, Teper A. Dupilumab Efficacy and Safety in Moderate-to-Severe Uncontrolled Asthma. N Engl J Med. 2018 Jun 28;378(26):2486-2496. doi: 10.1056/NEJMoa1804092. Epub 2018 May 21.

    PMID: 29782217RESULT

  • Snelder SM, Weersink EJM, Braunstahl GJ. 4-month omalizumab efficacy outcomes for severe allergic asthma: the Dutch National Omalizumab in Asthma Registry. Allergy Asthma Clin Immunol. 2017 Jul 26;13:34. doi: 10.1186/s13223-017-0206-9. eCollection 2017.

    PMID: 28769983RESULT

  • van Toor JJ, van der Mark SC, Kappen JH, In 't Veen JCCM, Braunstahl GJ. Mepolizumab add-on therapy in a real world cohort of patients with severe eosinophilic asthma: response rate, effectiveness, and safety. J Asthma. 2021 May;58(5):651-658. doi: 10.1080/02770903.2020.1723623. Epub 2020 Feb 12.

    PMID: 31999203RESULT

  • Upham JW, Le Lievre C, Jackson DJ, Masoli M, Wechsler ME, Price DB; Delphi Panel. Defining a Severe Asthma Super-Responder: Findings from a Delphi Process. J Allergy Clin Immunol Pract. 2021 Nov;9(11):3997-4004. doi: 10.1016/j.jaip.2021.06.041. Epub 2021 Jul 13.

    PMID: 34271216RESULT

MeSH Terms

Conditions

Asthma

Interventions

dupilumab

Condition Hierarchy (Ancestors)

Bronchial DiseasesRespiratory Tract DiseasesLung Diseases, ObstructiveLung DiseasesRespiratory HypersensitivityHypersensitivity, ImmediateHypersensitivityImmune System Diseases

Study Officials

  • Jursica Mills, MSc

    Wetenschapsbureau FGV

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

April 2, 2022

First Posted

April 18, 2022

Study Start

April 1, 2021

Primary Completion

October 30, 2022

Study Completion

December 31, 2022

Last Updated

September 21, 2023

Record last verified: 2023-09

Locations

NL(1)