A Phase 2 Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib (LOU064) in Adult Patients With Papulopustular Rosacea (PPR)

A Multicenter, Randomized, Double-blind, Placebo-controlled Phase 2 Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib (LOU064) in Adult Patients With Papulopustular Rosacea (PPR)

This Phase 2 study aims to evaluate whether Bruton's tyrosine kinase (BTK) inhibition with remibrutinib can produce a clinically meaningful reduction in inflammatory lesions in adults with moderate-to-severe papulopustular rosacea, while also assessing safety and tolerability of remibrutinib in this indication.

Study Overview

• Status: Not yet recruiting
• Conditions: Papulopustular Rosacea
• Intervention / Treatment: Drug: Placebo; Drug: LOU064

Detailed Description

This is a multicenter, randomized, participant and Investigator-blinded, placebo-controlled, parallel-group Phase 2 study designed to evaluate the efficacy, safety, and tolerability of remibrutinib in adults with moderate-to-severe PPR. Following a screening period of up to 30 days, which can be extended by a further 2 weeks only to allow washout from rosacea treatments and other systemic therapies as specified in the prohibited medication section, eligible participants will be randomized at baseline to receive either remibrutinib or matching placebo for a 16-week double-blind treatment phase. A safety follow up visit will occur approximately 30 days after the final dose.

• Study Type: Interventional
• Enrollment (Estimated): 80
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: 1-888-669-6682; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals; Phone Number: +41613241111

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria:

• Signed informed consent must be obtained prior to participation in the study.
• Adult ≥18 years with a clinical diagnosis of PPR.
• Moderate-to-severe disease defined by a modified Investigator's Global Assessment (IGA) score of 3 or 4
• The presence of 15 - 60 inflammatory (papular/pustular, max. 2 nodular) facial lesions at screening, with at least 15 lesions present at Day 1 (Baseline).
• Completed requisite washout of systemic antibiotics (30 days) and other prohibited systemic therapies before randomization.
• Willingness to refrain from initiating treatments or undergoing procedures that target or impact PPR during the double-blind period, and to use only protocol-allowed products.

Key Exclusion Criteria:

• Presence of more than 2 nodular inflammatory lesions
• Any active facial dermatoses or skin disease or condition that may interfere with assessment of PPR (e.g., seborrheic dermatitis, perioral dermatitis, acne, acneiform eruptions from biologic medications, steroid-induced dermatitis resembling rosacea or acne).
• History of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical classes
• Use of biologics within five half-lives prior to screening or until the expected pharmacodynamic (PD) effect has returned to baseline, whichever is longer; or longer if required by local regulations
• Use of small molecules and/or immunosuppressants that are not corticosteroids within 5 half-lives or within 30 days prior to screening, whichever is longer; or longer if required by local regulations
• Any use of systemic corticosteroids, systemic antibiotics, or topical treatments (including corticosteroids, antibiotics, ivermectin, azelaic acid, or metronidazole) within 30 days prior to randomization, or any planned use of these agents during the study treatment period.
• History of live attenuated vaccine within 6 weeks prior to randomization or requirement to receive these vaccinations at any time while on study treatment.
• Use, planned use, or failure to meet the protocol-defined washout periods for prohibited therapies. In particular, patients with pretreatment with remibrutinib or another BTK-inhibitor within 4 months prior to randomization.

Other protocol-defined inclusion/exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; Matching placebo	Drug: Placebo; Matching placebo
Experimental: LOU064; LOU064 administered by oral route	Drug: LOU064; LOU064 administered by oral route; Other Names: Remibrutinib

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Absolute change from baseline in facial inflammatory lesion count	The facial inflammatory lesion count is defined as the sum of papules, pustules, and nodules present on the face. Lesions are visually counted across the full facial area (forehead, cheeks, nose, chin). A negative value indicates a reduction in lesions (clinical improvement), as lower counts reflect less inflammatory activity.	Baseline, Week 16

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants with Investigator's Global Assessment (IGA,modified scale without erythema) grade 0 or 1, with at least 2 grade reduction from baseline	The Investigator's Global Assessment is a clinician-reported outcome using a 5-point ordinal scale (0-4). Higher scores indicate more severe disease; lower scores indicate improvement. 0 = Clear; = Near clear; = Mild; = Moderate; = Severe	Baseline, Week 16
Percentage change from baseline in facial inflammatory lesion count	The facial inflammatory lesion count is defined as the sum of papules, pustules, and nodules present on the face. Lesions are visually counted across the full facial area (forehead, cheeks, nose, chin). Lower values indicate fewer lesions. A reduction from baseline represents clinical improvement.	Baseline, Week 16

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Estimated): July 29, 2026
• Primary Completion (Estimated): December 7, 2027
• Study Completion (Estimated): December 7, 2027

Study Registration Dates

• First Submitted: June 26, 2026
• First Submitted That Met QC Criteria: June 26, 2026
• First Posted (Actual): July 2, 2026

Study Record Updates

• Last Update Posted (Actual): July 2, 2026
• Last Update Submitted That Met QC Criteria: June 26, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: tolerability; safety; Papulopustular Rosacea; efficacy; PPR; Ph2
• Additional Relevant MeSH Terms: Skin Diseases; Skin and Connective Tissue Diseases; Rosacea; Primary hyperoxaluria type 2; remibrutinib
• Other Study ID Numbers: CLOU064Q12201; 2025-524496-23-00 (Other Identifier: EU CTIS)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No