NCT05032157

A Phase 3 Study of Efficacy and Safety of Remibrutinib in the Treatment of CSU in Adults Inadequately Controlled by H1-antihistamines

A Multicenter, Randomized, Double-blind, Placebo-controlled Phase 3 Study of Remibrutinib (LOU064) to Investigate the Efficacy, Safety and Tolerability for 52 Weeks in Adult Chronic Spontaneous Urticaria (CSU) Patients Inadequately Controlled by H1-antihistamines

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 455 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to establish the efficacy, safety, and tolerability of Remibrutinib 25 mg b.i.d. in adult patients suffering from chronic spontaneous urticaria (CSU) inadequately controlled by second generation H1-antihistamines (H1-AHs) in comparison to placebo.

Detailed description

The study consisted of four periods, the total study duration was up to 60 weeks: Screening period of up to 4 weeks, Double-blind placebo-controlled treatment period of 24 weeks, Open-label treatment period with Remibrutinib period of 28 weeks, and treatment free follow-up period of 4 weeks. The design of this study was a replicate of another Phase III study, CLOU046A2301 (NCT05030311). The study population consisted of female and male adult patients with CSU inadequately controlled by second generation H1-AHs at least at a locally label approved dose. All patients were on a stable, locally label approved dose of a second generation H1 AH (background therapy) throughout the entire study (starting a minimum of 7 days prior to randomization until the end of the study). To treat unbearable symptoms of CSU, patients were allowed to use another second generation H1-AH on an as-needed basis (rescue therapy). Eligible patients were randomly assigned to the treatment arms in a 2:1 ratio to remibrutinib or placebo arm (300 in the remibrutinib arm and 150 in placebo arm) and stratified based on prior exposure to anti-IgE biologics for CSU and geographic region. An extension Phase IIIb study, CLOU064A2303B (NCT05513001), was initiated to allow CLOU064A2302 eligible patients to roll over after completion of the open-label treatment period. There were two distinct testing strategies (scenario 1 with Weekly Urticaria Activity Score (UAS7) as the primary efficacy endpoint and scenario 2 with Weekly Itch Severity Score (ISS7) and Weekly Hives Severity Score (HSS7) as the co-primary efficacy endpoints) based on two primary objective scenarios related to regional regulatory precedent and Health Authorities' feedback.

Conditions

• Chronic Spontaneous Urticaria

Interventions

Type	Name	Description
DRUG	LOU064 (blinded)	LOU064 (blinded) active treatment
DRUG	Placebo	Placebo
DRUG	LOU064 (open-label)	LOU064 (open-label) active treatment

Timeline

• Start date: 2021-12-01
• Primary completion: 2023-12-18
• Completion: 2024-01-05
• First posted: 2021-09-02
• Last updated: 2025-04-08
• Results posted: 2024-11-01

Locations

122 sites across 18 countries: United States, Austria, Brazil, Canada, China, Denmark, Germany, India, Malaysia, Poland, Russia, Slovakia, South Africa, Switzerland, Taiwan, Thailand, United Kingdom, Vietnam

Regulatory

• FDA-regulated drug study