Remibrutinib in Real-world Clinical Practice - a Germany Sub-study (REASSERT)

N/ANot Yet RecruitingNCT07642557

Novartis Pharmaceuticals470 enrolled

Overview

Prospective, non-interventional study in patients with CSU where the treatment decision prior enrolment has been made to 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.

This is a prospective, non-interventional study in patients with CSU where the treatment decision prior enrolment has been made to 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. This local Germany study is part of an umbrella design which brings the evidence needs from multiple countries under the REASSERT global program (CLOU064A2402).

Study Type

OBSERVATIONAL

Enrollment

470

Conditions

Chronic Spontaneous Urticaria

Eligibility

Sex: ALLMin age: 18 YearsMax age: 100 Years

Medical Language ↔ Plain English

Inclusion Criteria: 7.2.1 Inclusion criteria Patients must meet all the following criteria to be eligible for inclusion in this study. 1. Patients with a confirmed diagnosis of primary CSU by the treating physician. 2. Aged at least 18 years on the date of enrollment. 3. Written informed consent of the patient to participate in the study and willingness to complete full follow-up period of 24 months. 4. Cohort-specific observational inclusion criteria: 1. 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 enrollment) to switch to remibrutinib treatment as per local label. 2. Cohort3: Any other treatment received in addition to H1-AH, any time during patients' CSU treatment history, with decision (independent of study enrollment) 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 3 weeks during treatment history, they will be included in cohort 3. Exclusion Criteria: Patients are excluded if they meet any of the following criteria: 1. Simultaneous participation in any investigational trial or simultaneous participation in another Novartis-sponsored non-interventional study with remibrutinib. 2. Patients within the safety follow-up phase of a previous interventional or non-interventional study. 3. Patients who received remibrutinib at any time in the past. 4. Patients not capable or willing to continuously provide ePRO/eDiary data via electronic means throughout the duration of the study. 5. Patients who are treated with remibrutinib outside of the local label.

Outcomes

Primary Outcomes

Achievement of well-controlled disease (UCT7 ≥ 12)

Achievement of well-controlled disease (UCT7 ≥ 12). 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)

Time frame: 12 weeks after initiating remibrutinib treatment

Secondary Outcomes

Number and proportion of participants with well-controlled disease and complete disease

Proportion of patients with UCT score ≥12 and UAS7 score ≤6 (well controlled disease) and UCT=16 and UAS7=0 (complete control). Urticaria Control Test (UCT) consists of 4 questions scored 0 (very much) to 4 (not at all), with a total score range of 0 to 16. High scores indicates better disease control. Urticaria Activity Score over 7 days (UAS7) is based on 2 daily questions, with daily score of 0 to 6 and weekly score of 0 to 42. Lower scores suggests urticaria free and well controlled urticaria.

Time frame: Up to 24 months

Change from baseline (CFB) in UCT score

Change in UCT score over time. Urticaria Control Test (UCT) consists of 4 questions scored 0 (very much) to 4 (not at all), with a total score range of 0 to 16. Highest scores indicates better disease control. The study is not powered to detect any specific difference and the purpose is to estimate any observed difference descriptively.

Time frame: Up to 24 months

Change from Baseline in UAS7 score ≥ minimal important difference

Urticaria Activity Score over 7 days (UAS7) is based on 2 daily questions, with daily score of 0 to 6 and weekly score of 0 to 42. Lower scores suggests urticaria free and well controlled urticaria. The study is not powered to detect any specific difference and the purpose is to estimate any observed difference descriptively.

Time frame: Up to 24 months

Change from baseline in UAS7 score

Central Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111 novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Data from ClinicalTrials.gov

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Time frame: Up to 24 months

Number of weeks without angioedema

Number of weeks without angioedema evaluated using application entries.

Time frame: Up to 24 months

Change in Angioedema Control Test (AECT)

The AECT consists of 4 questions scored 0 (very much) to 4 (not at all) that measures angioedema control over recent weeks, with a total score ≥10 indicating well-controlled disease

Time frame: Baseline, up to 24 months

Number of participants with rescue medication requirement

Time frame: Up to 24 months

Characterize CSU relapse

CSU relapse by frequency and presentation

Time frame: Up to 24 months

UCT7 score

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).

Time frame: Up to 12 weeks

UAS7 score

Urticaria Activity Score over 7 days (UAS7) is based on 2 daily questions, with daily score of 0 to 6 and weekly score of 0 to 42. Lower scores suggests urticaria free and well controlled urticaria. The study is not powered to detect any specific difference, descriptive analysis of difference over time will be presented.

Time frame: Up to 12 weeks

Number of participants with administration of remibrutinib according to label

Time frame: Up to 24 weeks

Number of participants with usage of concomitant sgH1-AHs and additional prescribed CSU treatments

Descriptive analysis of concomitant sgH1-AHs

Time frame: Up to 24 weeks

Number of participants with administration of CSU-related treatment(s) outside of local and/or international guidelines

Time frame: 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

Composite analytical endpoint comprising the proportion of participants non-adherent to local/international CSU treatment guidelines, and associated impact of patient (WPAI, DLQI), disease control (UAS7, UCT/UCT7), treatment patterns (treatment switches, discontinuations, dose modifications), and healthcare resource utilisation (CSU-related inpatient hospitalizations, length of stay, emergency room visits, outpatient visits) during early (up to Week 12) and long-term (up to Month 24) follow-up

Time frame: Up to 24 months

Time without escalation, with UCT<12 or UAS7>6

Time in days/months without escalation while UCT score is below 12 OR UAS7 score above 6. Urticaria Control Test consists of 4 questions, score below 12 indicates poor disease control. Urticaria Activity Score over 7 days is based on 2 daily questions with a maximum daily score of 6 and weekly total score up to 42. Lower scores indicates better urticaria control.

Time frame: Up to 24 months

Quality of life measured through DLQI score

Dermatology Life Quality Index (DLQI) score consists of 10 questions scored from 0 (no impact at all) to 3 (very much). Total score ranges from 0 to 30, with lower scores showing lowest impact of disease on quality of life

Time frame: Up to 24 months

Quality of life evaluated through Chronic Urticaria Quality of Life Questionnaire (CU-Q2oL) questionnaire scores

Change over time in Chronic Urticaria Quality of Life Questionnaire (CU-Q2oL) questionnaire. CU-Q2oL consists of 20 items rated from 0 (never) to 5 (very often). Total score ranges from 0 to 25, with lower scores showing better urticaria control. The study is not powered to detect any specific difference, descriptive analysis of difference over time will be presented.

Time frame: Up to 24 months

Quality of life measured through Sleep interference score

Sleep interference will be evaluated though bespoke question. Maximum score along 7 days ranges from 0 to 21, lower scores showing lowest interference of CSU with sleep.

Time frame: Up to week 12

Quality of life measured through Hospital anxiety and depression scale (HADS)

Quality of life will be evaluated through HADS measuring anxiety and depression components, as well as change from baseline in HADS score. Hospital Anxiety and Depression Score (HADS) is a 14 item questionnaire, 7 item rating anxiety and 7 depression. Responses are rated from 0 (never) to 3 (almost all time) at each question. Total score for anxiety and total score for depression range from 0 to 21, higher scores showing higher anxious/depressive symptomatology. Descriptive analysis will be presented

Time frame: Up to 24 months

Number of events related to CSU or Health Care Resource utilisation

Health Care resource utilisation (HCRU) will be evaluated based on quarterly and annualized number of health care resource utilisation related to CSU.

Time frame: Up to 24 months

Work productivity and activity impairment (WPAI-CU) score

Work productivity assessed through WPAI questionnaire which consists of 6 questions, used to calculate 4 types of scores, absenteeism (work time missed), presenteeism (impairment at work/reduced on the job effectiveness), work productivity loss (overall work impairment), and activity impairment. The scores are expressed in percentage with higher numbers indicating greater impairment and less productivity at work.

Time frame: Up to 24 months

Incidence of AEs

Incidence of AEs, including serious AEs in patients who initiated remibrutinib

Time frame: Up to 24 months