Clinical trial NCT06683261: Real-life Evaluation of the Efficacy of Biologicals in Chronic Rhinosinusitis With Nasal Polyposis (CRSwNP)
Official title: Real-life Evaluation of the Efficacy of Biologicals in CRSwNP
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Chronic Rhinosinusitis With Nasal Polyps (CRSwNP)
Chronic Rhinosinusitis (CRS)
Keywords: biologicals
CRSwNP
real-world data study
mepolizumab
omalizumab
dupilumab
Lead sponsor: Universitaire Ziekenhuizen KU Leuven
Sponsor class: OTHER
First posted: 2024-11-12
Enrollment: 300
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with CRSwNP that are prescribed a biological in the outpatient clinic of several ENT departments across Belgium

Brief summary: The goal of this observational study is to learn more about the real-world efficacy of biologics in patients with chronic rhinosinusitis with nasal polyposis (CRSwNP). All patients who are 18 years or older, who have CRSwNP and are eligible for reimbursement of a biological for the indication of CRSwNP can be included.

The main question is the efficacy of biologics in real life after 24 weeks. The main focus are patient reported outcomes measured via several questionnaires and the nasal polyp score, scored with nasal endoscopy.

Detailed description: Chronic rhinosinusitis with nasal polyps (CRSwNP) has a prevalence of 5-10% in adults. It is often associated with other comorbidities, such as asthma (30-70% of CRSwNP patients) and aspirin exacerbated respiratory disease (AERD) (16% of CRSwNP patients) leading to a significant reduced quality of life (QoL). CRSwNP is diagnosed in patients with nasal polyps who have 2 or more of the following symptoms for more than 12 weeks:

* Nasal blockage or nasal congestion
* Runny nose or postnasal drip
* Impaired sense of smell
* Facial pain/pressure. Nasal rinses, intranasal or systemic corticosteroids, long-term antibiotics, and sinus surgery are the current standard of care. However, many patients fail to achieve complete therapeutic benefit and relapse after time, even after surgery. Moreover, oral corticosteroids are associated with significant side effects, and repeated sino-nasal surgery becomes progressively more complex with higher risk of complications. Patients with CRSwNP and most patients with asthma share a common type 2 inflammatory response, characterised by elevated levels of interleukin (IL)-4, IL-5, IL-13, eosinophils, T helper 2 (Th2)cells, and type 2 innate lymphoid cells. In addition, locally produced immunoglobulin E (IgE) is able to activate mast cells and induce local inflammation in CRSwNP.

Biologicals are a specific kind of treatment with recombinant DNA-derived humanized monoclonal antibody that selectively binds specific targets in the inflammatory cascade which contributes to the pathophysiology of CRSwNP. Different biologicals have already been reimbursement for the treatment of Asthma for years, slowly these biologicals start to get their approval and reimbursement for CRSwNP. In Belgium (omalizumab (Xolair®), mepolizumab (Nucala®) and dupilumab (Dupixent ®) are reimbursed for patients with CRSwNP. Their efficacy has been demonstrated through large double-blind placebo-controlled clinical studies. However, until now only very limited reports on real-world data regarding this therapy have been published. This real-world data is important because it enables us to go beyond data gathered throughout a traditional randomised controlled trial (RCT). Traditional RCTs gather data from a controlled sample population with limited comorbidities and concomitant medications, who are likely to be compliant with the study requirements, whereas in real life patients might have poorer performance status and compliance and consist of a higher proportion of elderly patients.

Therefore, this real-world data study aims to investigate how clinical outcomes of biologic therapy in real-world application (real-world efficacy) corresponds to outcomes in clinical trials (efficacy) and to look into factors that might explain an efficacy gap.

Eligibility criteria: Inclusion Criteria:

1. Participant must be at least 18 years of age at the time of signing the informed consent.
2. Capable of giving signed informed consent.
3. Participants should have CRSwNP.
4. Participants should fulfil the reimbursement criteria for their prescribed biological (omalizumab, mepolizumab and/or dupilumab).

Exclusion Criteria:

1. Patients treated with other biological therapies in the 3 months prior to the current biological (this is not applicable to patients who are in the trial and switch biological).

Primary outcomes:
measure: To evaluate the effect of biologicals (omalizumab, mepolizumab and dupilumab) on the nasal congestion score (NCS).
timeFrame: From enrollment until 3 years of treatment
Secondary outcomes:
measure: To evaluate the effect of biologicals (omalizumab, mepolizumab and dupilumab) on the nasal polyp score (NPS).
timeFrame: From enrollment until 3 years of treatment
measure: To evaluate the effect of biologicals (omalizumab, mepolizumab and dupilumab) on the Visual analogue scale (VAS).
timeFrame: From enrollment until 3 years of treatment
measure: To evaluate the effect of biologicals (omalizumab, mepolizumab and dupilumab) on the Sino-Nasal Outcome Test-22 items (SNOT-22).
timeFrame: From enrollment until 3 years of treatment
measure: To compare the effect of biologicals (omalizumab, mepolizumab and dupilumab) on the asthma control questionnaires 5-item version(ACQ-5).
timeFrame: From enrollment until 3 years of treatment
measure: To compare the effect of biologicals (omalizumab, mepolizumab and dupilumab) on the asthma quality of life questionnaire (AQLQ).
timeFrame: From enrollment until 3 years of treatment
measure: To compare the effect of omalizumab, mepolizumab and dupilumab on the sense of smell with the short questionnaire of olfactory disorders: negative statements (sQOD-NS).
timeFrame: From enrollment until 3 years of treatment
measure: To evaluate patient's adherence to their biological add-on treatment.
timeFrame: form enrollment until 3 years of treatment
Other outcomes:
measure: To characterise non-responders to any biological treatment.
timeFrame: from enrolment until 3 years of treatment
Locations:
city: Aalst
country: Belgium
facility: Onze Lieve Vrouw hospital Aalst
city: Bruges
country: Belgium
facility: General hospital Sint-Jan Bruges
city: Bruxelles
country: Belgium
facility: University hospital Saint-Luc
city: Edegem
country: Belgium
facility: University hospital of Antwerp
city: Genk
country: Belgium
facility: Hospital Zuid-Oost Limburg
city: Jette
country: Belgium
facility: University Hospital Brussels
city: Leuven
country: Belgium
facility: University hospitals Leuven
city: Liège
country: Belgium
facility: CHU de Liège
Study officials:
affiliation: Universitaire Ziekenhuizen KU Leuven
name: Peter Hellings, MD,PhD
role: PRINCIPAL_INVESTIGATOR
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