Remibrutinib in Real-world Clinical Practice: a Prospective, Multi-country, Non-interventional, Effectiveness and Safety Study
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Novartis Pharmaceuticals
- Enrollment
- 3,280
- Locations
- 1
- Primary Endpoint
- UCT7 score
Study Overview
Brief Summary
Prospective, multi-country, non-interventional study in patients with CSU where the treatment decision prior enrolment has been made to either escalate current sgH1-AHs treatment or escalate/switch current treatment to remibrutinib. The primary aim of this study is to gather real-world effectiveness and safety data for remibrutinib, a new treatment option, covering a broader, real-world clinical practice population.
Detailed Description
This is a prospective, multi-country, non-interventional study in patients with CSU where the treatment decision prior enrolment has been made to either escalate current sgH1-AHs treatment or escalate/switch current treatment to remibrutinib. The primary aim of this study is to gather real-world effectiveness and safety data for remibrutinib, covering a broader, real-world clinical practice population. The study employs an umbrella design which brings the evidence needs from multiple countries under the REASSERT global program. Countries will generate local protocol to be used in their country; the local study documentation will be a minimised version of the global document, modified to align with country's evidence needs, given local disparities in treatment guidelines, access, physician type and ePROs/eDiaries used. To achieve the core objectives the observation needs to include either the Urticaria Control Test (UCT) or Urticaria Activity Score over 7 days (UAS7) and the Dermatology Life Quality Index (DLQI). Data from all countries will be pooled and analyzed globally. In certain instances, some modifications of the global protocol may be permitted. For example, in countries where prospective monitoring of off-label antihistamines is prohibited, cohort 1 will not be enrolled.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Patients with a confirmed diagnosis of primary CSU by the treating physician.
- •Aged at least 18 years on the date of enrolment.
- •Written informed consent of the patient to participate in the study (according to country specifications) and willingness to complete full follow-up period of 24 months.
- •Cohort-specific observational inclusion criteria:
- •Cohort 1: Inadequate control of CSU despite licensed dose of sgH1-AH (no other pre-treatments permitted) and decision (independent of study enrolment) to escalate sgH1-AH treatment.
- •Cohort 2: Inadequate control of CSU despite licensed dose or escalated sgH1-AH(s) (no other pre-treatment with exception of first generation H1-AH permitted) with decision (independent of study enrolment) to switch to remibrutinib treatment as per local label.
- •Cohort 3: Any other treatment received in addition to H1-AH, any time during patients' CSU treatment history, with decision (independent of study enrolment) to switch to remibrutinib treatment as per local label. Note, occasional steroid rescue medication is out of scope for cohort definition. If a patient had been on continuous steroids for at least three weeks during treatment history, they will be included in cohort
- •Note: Candidate patients must not have initiated the next escalated treatment step (i.e. up dosed AH for cohort 1, or remibrutinib for cohorts 2 and 3) prior to their enrolment to ensure the baseline visit captures their clinical status before treatment escalation.
Exclusion Criteria
- •Currently enrolled in a clinical trial or on any experimental treatment.
- •Patients within the safety follow-up phase of a previous interventional or non-interventional study.
- •Patients who received remibrutinib as an investigational medical product during a remibrutinib interventional study or MAP/PSDS at any time in the past.
- •Patients not capable or willing to continuously provide ePRO/eDiary data via electronic means throughout the duration of the study.
- •Patients who are treated with remibrutinib outside of the local label.
Arms & Interventions
Cohort 1
Inadequate control of CSU despite licensed dose of sgH1-AH (no other pre-treatments permitted) and decision to escalate sgH1-AH treatment
Cohort 2
Inadequate control of CSU despite licensed dose or escalated sgH1-AH(s) (no other pre-treatment with exception of first generation H1-AH permitted) with decision to switch to remibrutinib treatment as per local label
Cohort 3
Any other treatment received in addition to H1-AH, any time during patients' CSU treatment history, with decision to switch to remibrutinib treatment as per local label
Outcomes
Primary Outcomes
UCT7 score
Time Frame: 12 weeks after initiating remibrutinib treatment
Urticaria Control Test is looking retrospectively for 7 days (UCT7) and consists of 4 questions. Each question is scored 0-4, with a total score range of 0 (poor control) to 16 (well controlled).
UAS7 score
Time Frame: 12 weeks after initiating remibrutinib treatment
Urticaria Activity Score over 7 days UAS7 is based on 2 daily questions scored 0-3 per day, ranging from 0 (complete control) to 42 (poor control)
Secondary Outcomes
- Number and proportion of participants with well-controlled disease and complete disease(Up to 24 months)
- Change from baseline (CFB) in UCT score(Up to 24 months)
- Change from Baseline in UAS7 score ≥ minimal important difference(Up to 24 months)
- Change in angioedema activity score (AAS7) from baseline(Up to 24 months)
- Number of weeks without angioedema(Up to 24 months)
- Number of participants with rescue medication requirement(Up to 24 months)
- Characterize CSU relapse(Up to 24 months)
- UCT score(Up to 24 months)
- UAS7 score(Up to 24 months)
- Change in AECT from baseline(Up to 12 weeks)
- Number of participants with administration of remibrutinib as monotherapy(Up to 24 months)
- Monotherapy duration(Up to 24 months)
- Number of participants with Administration of remibrutinib as monotherapy with "on demand" sgH1-AH(Up to 24 months)
- Duration of monotherapy with "on demand" sgH1-AH(Up to 24 months)
- Number of participants with usage of concomitant sgH1-AHs and additional prescribed CSU treatments(Up to 24 months)
- Number of participants with administration of CSU-related treatment(s) outside of local and/or international guidelines(Up to 24 months)
- Demonstrate relationship between non-adherence to local/international guidelines and impact on patient, disease control, treatment patterns and HCRU during early and long-term follow-up(Up to 24 months)
- Delay of treatment escalation contrary to local and/or international guidelines when UCT <12 or UAS>6(Up to 24 months)
- Time without escalation, with UCT<12 or UAS7>6(Up to 24 months)
- Number of participants with escalation to remibrutinib(Up to 24 months)
- Number of participants with administration of above the licensed dose of sgH1-AH prior to remibrutinib switch(Up to 24 months)
- Number of sgH1-AH treatment escalations and switches prior to remibrutinib escalation(Up to 24 months)
- Demonstrate benefit of early vs late treatment escalation to remibrutinib(Up to 24 months)
- Proportion of patients with uncontrolled angioedema escalated to remibrutinib(Up to 24 months)
- Proportion of patients requiring corticosteroids prior to switch to remibrutinib(Up to 24 months)
- Quality of life measured through DLQI score(Up to 24 months)
- Quality of life evaluated through Chronic Urticaria Quality of Life Questionnaire (CU-Q2oL) questionnaire scores(Up to 24 months)
- Quality of life measured through Sleep interference score(Up to week 12)
- Quality of life measured through Hospital anxiety and depression scale (HADS)(Up to 24 months)
- Number of events related to CSU or Health Care Resource utilisation(Up to 24 months)
- Work productivity and activity impairment (WPAI-CU) score(Up to 24 months)
- Incidence of AEs(Up to 24 months)