Clinical trial NCT06566885: Real-world Study Assessing Efficacy of TezepeLumaB in Patients With Severe Asthma Regardless of Phenotype in Russia
Official title: Multicentre, Single Arm, Non-interventional, Observational, Prospective Study to Assess Demographic Characteristics, Burden of Disease and Short-term Patient Reported Outcomes on Symptom Relief in Severe Asthma Patients Aged Older Than 12 Qualifying for Treatment With Tezepelumab in Russia
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Severe Asthma
Keywords: tezepelumab
severe asthma
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
First posted: 2024-08-22
Enrollment: 110
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
Sampling method: PROBABILITY_SAMPLE
Study population: Participants of both sexes, aged 12 years or older with severe uncontrolled asthma despite medium or high doses of ICS, who have commenced/will commence treatment with tezepelumab as determined by their routine clinical care.

The study population is planned to comprise approximately 110 participants in approximately 20 sites. Eligible patients will be enrolled consecutively at each site with a maximum quota per site to minimize selection bias at each site

Brief summary: ELBRUS is a 12-month (52-week), multi-centre, prospective, non-comparative and non-interventional (observational), post-reimbursement real-world evidence study that will assess patient-reported outcomes after tezepelumab treatment initiation in participants with severe asthma in Russia.

Detailed description: This is a multi-centre, retrospective-prospective, non-comparative and non-interventional (observational) cohort study involving primary and secondary data collection within real-world settings of participants who initiate tezepelumab for treatment of severe uncontrolled asthma. Participants of the study will be the patients eligible for tezepelumab treatment based on the assessment in accordance with the approved product Summary of Product Characteristics (SmPC) in Russia. The administration of tezepelumab will be independent of this study (i.e., a decision of tezepelumab initiation is based on the physician's choice and regulatory and clinical features, not on recruitment/participation in the study). Tezepelumab is indicated as an add-on maintenance treatment for severe asthma, therefore, patients will continue background asthma therapy throughout the study, irrespective of their decision to participate in this study or not.

110 eligible participants of both sexes, aged 12 years or older will be treated with tezepelumab available in the market and according to the Russian reimbursement policies in approximately 20 sites. In eligible participants who agree to take part in the study, the enrolment date is defined as the date of informed consent or assent. After enrolment and evaluation of inclusion/exclusion criteria, study participants will commence tezepelumab treatment as per the physician's decision and following the local product SmPC. The index date is defined as the date when participants have received the first dose of tezepelumab. The enrolment period is the period between enrolment date and index date. Additionally, participants may be enrolled in this study up to 4 weeks after the first dose of tezepelumab, but no longer, to avoid responder bias.

Participants will be followed for a maximum period of 52 weeks after index date, irrespective of treatment discontinuation. Patient-reported outcomes - the primary endpoint (ACQ-5) and SNOT-22 will be retrospectively collected during enrolment for all patients (i.e. the most recent available values in the 52 weeks prior to index date), and prospectively collected at suggested visits at Weeks 4, 12, 24, and 52 following index date. For patients who initiate treatment after being enrolled into the study the baseline value may be collected prospectively after enrolment before start of treatment.

The baseline period is defined as the 52 weeks prior to the index date. Outcomes of interest, such as severe asthma exacerbations, medication use, and healthcare resource utilization, will be collected during enrolment retrospectively for the baseline period (52 weeks prior to the index date) and then prospectively at Weeks 4, 12, 24, and 52 following index date.

Overall expected duration of the study (from the first patient inclusion to the last patient last visit) is about 2 years or until 110 eligible patients are included to the study and data on these patients are collected, whichever occurs first.

As an observational, this study does not imply any intervention into a routine clinical practice, including choice of treatment modality or additional diagnostic methods.

Eligibility criteria: Inclusion Criteria:

be eligible for enrolment into this study if all of the following criteria are met:

1. Male or female participants aged 12 years or older at the time of signing the ICF or assent.
2. Prescribed treatment with tezepelumab according to SmPC and local market reimbursement criteria. If a patient is enrolled to the study after tezepelumab initiation, a period between treatment initiation and enrolment should be no more than 4 weeks.
3. Diagnosis of asthma established for at least 52 weeks prior to tezepelumab initiation and symptoms confirmed by the Investigator not to be due to alternative diagnoses.
4. Received at least one prescription of medium or high doses of ICS during the 52 weeks prior to tezepelumab initiation, with medium or high doses of ICS defined according to the GINA 2022 (see below Note #1).
5. Use of additional asthma maintenance controller medication(s) in addition to ICS (e.g., LABA, leukotriene receptor inhibitors, theophylline, LAMA, and cromones) for at least 52 weeks prior to tezepelumab initiation. The additional maintenance controller medication may be contained in a combination product (e.g., ICS/LABA).
6. Documented history of at least 2 severe asthma exacerbations during the 52 weeks prior to tezepelumab initiation. For participants receiving prior biologic drugs for ≥ 8 months, at least 1 severe exacerbation must have occurred on prior biologic treatment. Participants are excluded if, in the opinion of the Investigator, prior biologic treatment had provided significant clinical benefit in the past 52 weeks, despite the participant experiencing ≥ 2 severe exacerbations during 52 weeks prior to tezepelumab initiation.
7. Individuals with ACQ-5 score ≥ 1.5 (indicating inadequate asthma symptom control) at enrolment or up to 12 weeks before enrolment.
8. Currently receive care from specialist physicians (e.g., pulmonologists and/or allergists) at the Investigator's or sub-Investigator's site.
9. Provision of signed and dated written informed consent, including assent (informed consent for participants under 18 years old).
10. Participants are able to read, understand and complete the questionnaires required by the protocol.

Exclusion Criteria:

1. Any contraindication to tezepelumab as per the approved product SmPC in Russia or in the opinion of the Investigator.
2. Administration of concurrent biologic drug for asthma since the index date, except for stable allergen immunotherapy (defined as a stable dose and regimen at the time of enrolment). Enrolment of patients who were switched from other biologic(s) to tezepelumab is allowed, and an acceptable timeframe since the last prior asthma biologic drug is ≥ 30 days. The number of participants with prior biologic treatment (switching to tezepelumab) should be targeted at 20% or less at each site.
3. Participation in an observational study that might, in the Investigator's opinion, influence the assessment for the current study, or participation in an interventional clinical trial in the last 3 months.
4. Pregnancy or lactation period.

Primary outcomes:
measure: Change in ACQ-5 score from baseline
timeFrame: 1 year
Secondary outcomes:
measure: 1. ACQ-5 score at different points of time
timeFrame: 1 year
measure: 2. Proportion of participants with well-controlled asthma
timeFrame: 1 year
measure: 3. Proportion of participants with ACQ-5 response
timeFrame: 1 year
measure: 4. Median time to ACQ-5 response
timeFrame: 1 year
measure: 5. Pre- and post-bronchodilator forced expiratory volume in 1 second
timeFrame: 2 years
measure: 6. Pre- and post-bronchodilator forced vital capacity
timeFrame: baseline and at Weeks 4, 12, 24 and 52 following tezepelumab initiation
measure: 7. Pre-bronchodilator forced expiratory flow
timeFrame: baseline and at Weeks 4, 12, 24 and 52 following tezepelumab initiation
measure: 8.1 Changes on pre- and post-bronchodilator FEV1 from baseline
timeFrame: baseline and at Weeks 4, 12, 24 and 52 following tezepelumab initiation
measure: 9. Proportion of pre- and post-bronchodilator FEV1 responders
timeFrame: 1 year
measure: 10.1 FRC
timeFrame: 1 year
measure: 11. Annualised rate of severe asthma exacerbations
timeFrame: 2 years
measure: 12.a Proportion of participants with reduced total number of severe asthma exacerbations at Week 52 following tezepelumab initiation compared with baseline
timeFrame: baseline during 52 weeks before tezepelumab initiation and up to 52 weeks following tezepelumab initiation
measure: 13. Change in annualised rate of severe asthma exacerbations from the baseline period to the follow-up period after tezepelumab initiation
timeFrame: baseline during 52 weeks before tezepelumab initiation and up to 52 weeks following tezepelumab initiation
measure: 14. Proportion of participants with 0, 1, 2, ≥3 severe asthma exacerbations
timeFrame: baseline during 52 weeks before tezepelumab initiation and up to 52 weeks following tezepelumab initiation
measure: 15. Cumulative days of severe asthma exacerbations
timeFrame: baseline during 52 weeks before tezepelumab initiation and up to 52 weeks following tezepelumab initiation
measure: 16. Proportion of participants with any SCS use
timeFrame: baseline during 52 weeks before tezepelumab initiation and up to 52 weeks following tezepelumab initiation
measure: 17. Proportion of participants with any reduction (≥ 25%, ≥ 50%, ≥ 75%, and 100% reduction) from baseline on cumulative SCS dose
timeFrame: during 52 weeks before tezepelumab initiation and up to 52 weeks following tezepelumab initiation
measure: 18. Change in median SCS dose from baseline
timeFrame: baseline during 52 weeks before tezepelumab initiation and up to 52 weeks following tezepelumab initiation
measure: 19. Proportion of participants with long-term (>30 consecutive days) SCS use
timeFrame: baseline during 52 weeks before tezepelumab initiation and up to 52 weeks following tezepelumab initiation
measure: 20. Number (proportion) of participants with asthma-related healthcare resource utilisation
timeFrame: baseline during 52 weeks before tezepelumab initiation and up to 52 weeks following tezepelumab initiation
measure: 21. Annualised rates of asthma-related hospitalisation, emergency calls (or emergency department visits), and unscheduled out-patient visits to a physician
timeFrame: baseline during 52 weeks before tezepelumab initiation and up to 52 weeks following tezepelumab initiation
measure: 22. Annualised rates of asthma-related scheduled physician visits or healthcare calls for asthma
timeFrame: baseline during 52 weeks before tezepelumab initiation and up to 52 weeks following tezepelumab initiation
measure: 23. Median overall duration (days) of asthma-related hospitalisations
timeFrame: baseline during 52 weeks before tezepelumab initiation and up to 52 weeks following tezepelumab initiation
measure: 24. Mean and median duration (days) of treatment with tezepelumab
timeFrame: Week 52
measure: 26. Median time (days) to tezepelumab discontinuation
timeFrame: Week 52
measure: 27. Proportion of participants discontinued tezepelumab and switched to other biologic drug for asthma treatment and reason(s)
timeFrame: Week 52
measure: 8.2 Changes on pre- and post-bronchodilator FVC from baseline
timeFrame: baseline and at Weeks 4, 12, 24 and 52 following tezepelumab initiation
measure: 8.3 Changes on pre-bronchodilator FEF from baseline
timeFrame: baseline and at Weeks 4, 12, 24 and 52 following tezepelumab initiation
measure: 12.b Proportion of participants with reduced total number of severe asthma exacerbations at Week 52 following tezepelumab initiation compared with baseline
timeFrame: up to 2 years
measure: 12.c Proportion of participants with reduced total number of severe asthma exacerbations at Week 52 following tezepelumab initiation compared with baseline
timeFrame: 2 years
measure: 10.2 RLV
timeFrame: 1 year
measure: 10.3 TLC
timeFrame: 1 year
Other outcomes:
measure: 1.1 Median time from index date to lung function response by FEV1
timeFrame: 1 year
measure: 2. Proportion of participants with sustained (12 weeks or longer) response by FEV1
timeFrame: 1 year
measure: 3.1 Median time from index date to spirometry and/or body plethysmography response
timeFrame: 1 year
measure: 4. Proportion of participants with sustained (12 weeks or longer) spirometry and/or body plethysmography response
timeFrame: 1 year
measure: 5. Proportion of participants with medications for asthma treatment by drug class
timeFrame: 2 years
measure: 6. Cumulative dose of ICS by each ICS medication
timeFrame: 2 years
measure: 7. Medication possession ratio (MPR)
timeFrame: Week 52
measure: 8. SNOT-22 total score
timeFrame: 1 year
measure: 9. Change on SNOT-22 total score from baseline
timeFrame: 1 year
measure: 10. Proportion of SNOT-22 responders (improvement of ≥ 8.9 in SNOT-22 total score from baseline) overall during the study and at different points of time
timeFrame: 1 year
measure: 11. A patient's body weight (kg) measured in participants with elevated BMI at enrolment
timeFrame: 1 year
measure: 12. Change in body weight from baseline calculated in participants with elevated BMI at enrolment
timeFrame: 1 year
measure: 13. Number and proportion of participants who achieved asthma clinical remission at Week 52
timeFrame: Week 52
measure: 1.2 Median duration of lung function response by FEV1 from index date
timeFrame: 1 year
measure: 3.2 Median duration of spirometry and/or body plethysmography response from index date (if available)
timeFrame: 1 year
Locations:
city: Ekaterinburg
country: Russian Federation
facility: Research Site
city: Kaliningrad
country: Russian Federation
facility: Research Site
city: Kemerovo
country: Russian Federation
facility: Research Site
city: Krasnodar
country: Russian Federation
facility: Research Site
city: Moscow
country: Russian Federation
facility: Research Site
city: Moscow
country: Russian Federation
facility: Research Site
city: Nizhny Novgorod
country: Russian Federation
facility: Research Site
city: Novosibirsk
country: Russian Federation
facility: Research Site
city: Orenburg
country: Russian Federation
facility: Research Site
city: Petrozavodsk
country: Russian Federation
facility: Research Site
city: Saint-Petersburg
country: Russian Federation
facility: Research Site
city: Stavropol
country: Russian Federation
facility: Research Site
city: Tomsk
country: Russian Federation
facility: Research Site
Study officials:
References: