--- title: "A Phase 2 Study to Evaluate the Safety and Efficacy of LOU064 in Patients With Moderate to Severe Sjögren's Syndrome" nct_id: NCT04035668 overall_status: TERMINATED phase: PHASE2 sponsor: Novartis Pharmaceuticals study_type: INTERVENTIONAL primary_condition: Sjögren Syndrome countries: United States, Australia, Belgium, Bulgaria, China, Denmark, Germany, Hungary, Spain, Switzerland, Taiwan, United Kingdom canonical_url: "https://parkinsonspathways.com/agent/trials/NCT04035668.md" clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT04035668" ct_last_update_post_date: 2023-01-30 last_seen_at: "2026-05-12T06:51:34.585Z" source: ClinicalTrials.gov (mirrored, no enrichment) --- # A Phase 2 Study to Evaluate the Safety and Efficacy of LOU064 in Patients With Moderate to Severe Sjögren's Syndrome **Official Title:** An Adaptive Phase 2 Randomized Double-blind, Placebo-controlled Multi-center Study to Evaluate the Safety and Efficacy of Multiple LOU064 Doses in Patients With Moderate to Severe Sjögren's Syndrome (LOUiSSe) **NCT ID:** [NCT04035668](https://clinicaltrials.gov/study/NCT04035668) ## Key Facts - **Status:** TERMINATED - **Why Stopped:** Sponsor's decision - **Phase:** PHASE2 - **Study Type:** INTERVENTIONAL - **Target Enrollment:** 73 - **Lead Sponsor:** Novartis Pharmaceuticals - **Conditions:** Sjögren Syndrome - **Start Date:** 2019-07-12 - **Completion Date:** 2021-11-23 - **CT.gov Last Update:** 2023-01-30 ## Brief Summary This was an adaptive design phase 2 study to establish safety and efficacy; and to characterize the dose-response of LOU064 in subjects with moderate to severe Sjögren's syndrome. LOU064 is an oral Bruton's tyrosine kinase (BTK) inhibitor. ## Detailed Description This study was planned as an adaptive Phase 2 randomized, double-blind, placebo-controlled, multi-center, integrated dose-ranging study to evaluate the safety and efficacy of multiple remibrutinib doses in patients with moderate to severe Sjögren's Syndrome. Of the initially planned two parts, only Part 1 of the study was conducted. In Part 1, the highest expected biologically active single dose of remibrutinib (100 mg) was tested in two different dosing regimens, a once daily dose (qd) or twice daily dose (bid), and compared to the placebo group. Each patient in Part 1 of the study underwent a screening period of up to 6 weeks, a treatment period of 24 weeks, and a follow-up period of 30 days post-treatment before the End of Study (EOS) visit. The total duration for each patient in the study, including Screening, was up to 35 weeks. For the treatment period, patients were randomized in a 1:1:1 ratio to one of the 3 treatment groups: remibrutinib 100 mg bid, remibrutinib 100 mg qd and placebo. ## Eligibility - **Minimum age:** 18 Years - **Maximum age:** 75 Years - **Sex:** ALL - **Healthy Volunteers:** No ``` Inclusion Criteria: * Diagnosis of SjS according to the 2016 ACR/EULAR criteria * Screening ESSDAI (based on weighted score) ≥ 5 derived from 8 domains * Screening ESSPRI ≥ 5 * Seropositive for anti-Ro/SSA antibodies at or within 3 months prior to screening * Unstimulated salivary flow \> 0 mL/min. Exclusion Criteria: * Sjögren's Syndrome overlap syndromes with another autoimmune disease as primary illness * DMARDs or kinase inhibitors within 3 months prior to baseline above certain doses OR maintained during study * Rituximab or other B cell depleting drug within 12 months of Screening . * Current use of prednisone or equivalent \> 15mg/d or dose change within 2 weeks prior to Screening * Use of medication known to cause, as a major side effect, dry mouth / eyes * HIV, Hepatitis C, Hepatitis B, known or suspected history of an ongoing, chronic or recurrent infectious disease such as tuberculosis ``` ## Arms - **Remibrutinib 100 mg bid** (EXPERIMENTAL) — Remibrutinib 100 mg twice daily (bid) - **Remibrutinib 100 mg qd** (EXPERIMENTAL) — Remibrutinib 100 mg once daily (qd) - **Placebo** (PLACEBO_COMPARATOR) — Placebo group ## Interventions - **Remibrutinib** (DRUG) — Remibrutinib 100 mg was administered orally as two 50 mg hard gelatin capsules. Patients in the remibrutinib 100 mg bid dose group took 2 capsules of active medication in the morning and 2 capsules of active medication in the evening. Patients in the remibrutinib 100 mg qd dose group took 2 capsules of active medication in the morning and 2 capsules of the placebo in the evening. - **Placebo** (DRUG) — Placebo was administered orally as two hard gelatin capsules. Patients in the placebo dose group took 2 capsules of placebo in the morning and 2 capsules of placebo in the evening. ## Primary Outcomes - **Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) Total Score at Week 24** _(time frame: Baseline, Week 24)_ — ESSDAI is a validated disease outcome measure for Sjögren's Syndrome. The instrument contains 12 organ-specific domains contributing to disease activity: constitutional, lymphadenopathy, glandular, articular, cutaneous, pulmonary, renal, muscular, peripheral nervous system, central nervous system, hematological and biological. For each domain, features of disease activity were scored according to their severity. These scores were summed across the 12 domains in a weighted manner to provide the total score. ESSDAI total score ranges from 0 to 123 with higher values indicating more disease activity. A negative change from baseline indicates improvement. The baseline value is defined as the last assessment performed prior to administration of the first dose of study treatment. A mixed effect model for repeated measurements (MMRM) was fitted to the changes from baseline in ESSDAI for all post-baseline time points up to Week 24. Values estimated from the model are presented in this table. ## Secondary Outcomes - **Change From Baseline in ESSDAI Total Score Over Time** _(time frame: Baseline, Week 2, Week 4, Week 8, Week 12, Week 16 and Week 20)_ - **Change From Baseline in EULAR Sjögren's Syndrome Patient Reported Index (ESSPRI) Total Score Over Time** _(time frame: Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24)_ - **Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-F) Total Score Over Time** _(time frame: Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24)_ - **Change From Baseline in EuroQual 5 Dimensions (EQ-5D) VAS Score Over Time** _(time frame: Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24)_ - **Change From Baseline in Physician Global Assessment Scale (PhGA) Score Over Time** _(time frame: Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24)_ - **Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs** _(time frame: From first dose of study treatment up 30 days after last dose (Week 29))_ - **Maximum Observed Blood Concentration (Cmax) of Remibrutinib at Week 4** _(time frame: pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4)_ - **Maximum Observed Blood Concentration (Cmax) of Remibrutinib at Week 24** _(time frame: pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 24)_ - **Time to Reach Maximum Observed Blood Concentration (Tmax) of Remibrutinib at Week 4** _(time frame: pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4)_ - **Time to Reach Maximum Observed Blood Concentration (Tmax) of Remibrutinib at Week 24** _(time frame: pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 24)_ - **Area Under the Blood Concentration-time Curve Within a Dosing Interval (Tau) at Steady-state (AUCtau) of Remibrutinib at Week 4** _(time frame: pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4)_ - **Area Under the Blood Concentration-time Curve Within a Dosing Interval (Tau) at Steady-state (AUCtau) of Remibrutinib at Week 24** _(time frame: pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 24)_ - **Area Under the Blood Concentration-time Curve From Time Zero to 4 Hours Post-dose (AUC0-4h) of Remibrutinib at Week 4** _(time frame: pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4)_ - **Area Under the Blood Concentration-time Curve From Time Zero to 4 Hours Post-dose (AUC0-4h) of Remibrutinib at Week 24** _(time frame: pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 24)_ - **Elimination Half-life (T1/2) of Remibrutinib at Week 4** _(time frame: pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4)_ - **Elimination Half-life (T1/2) of Remibrutinib at Week 24** _(time frame: pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 24)_ ## Locations (26) - Novartis Investigative Site, Boston, Massachusetts, United States - Novartis Investigative Site, Woodville, South Australia, Australia - Novartis Investigative Site, Hobart, Tasmania, Australia - Novartis Investigative Site, Clayton, Victoria, Australia - Novartis Investigative Site, Ghent, Belgium - Novartis Investigative Site, Sofia, Bulgaria - Novartis Investigative Site, Hefei, Anhui, China - Novartis Investigative Site, Nanjing, Jiangsu, China - Novartis Investigative Site, Chengdu, Sichuan, China - Novartis Investigative Site, Glostrup Municipality, Denmark - Novartis Investigative Site, Berlin, Germany - Novartis Investigative Site, Debrecen, Hungary - Novartis Investigative Site, Sabadell, Barcelona, Spain - Novartis Investigative Site, Vigo, Pontevedra, Spain - Novartis Investigative Site, Valencia, Valencia, Spain - Novartis Investigative Site, Barcelona, Spain - Novartis Investigative Site, Madrid, Spain - Novartis Investigative Site, Basel, Switzerland - Novartis Investigative Site, Lausanne, Switzerland - Novartis Investigative Site, Taichung, Taiwan ROC, Taiwan - Novartis Investigative Site, Kaohsiung City, Taiwan - Novartis Investigative Site, Taichung, Taiwan - Novartis Investigative Site, Taichung, Taiwan - Novartis Investigative Site, Liverpool, United Kingdom - Novartis Investigative Site, Swindon, United Kingdom - Novartis Investigative Site, Tyne and Wear, United Kingdom ## Recent Field Changes (last 30 days) - `outcomes.secondary` — added _(2026-05-12)_ - `status.overallStatus` — added _(2026-05-12)_ - `status.whyStopped` — added _(2026-05-12)_ - `status.primaryCompletionDate` — added _(2026-05-12)_ - `status.completionDate` — added _(2026-05-12)_ - `status.lastUpdatePostDate` — added _(2026-05-12)_ - `design.phases` — added _(2026-05-12)_ - `design.enrollmentCount` — added _(2026-05-12)_ - `eligibility.criteria` — added _(2026-05-12)_ - `eligibility.minAge` — added _(2026-05-12)_ - `eligibility.maxAge` — added _(2026-05-12)_ - `eligibility.sex` — added _(2026-05-12)_ - `outcomes.primary` — added _(2026-05-12)_ - `armsInterventions.arms` — added _(2026-05-12)_ - `armsInterventions.interventions` — added _(2026-05-12)_ - `sponsor.lead` — added _(2026-05-12)_ - `results.hasResults` — added _(2026-05-12)_ - `locations.novartis investigative site|boston|massachusetts|united states` — added _(2026-05-12)_ - `locations.novartis investigative site|woodville|south australia|australia` — added _(2026-05-12)_ - `locations.novartis investigative site|hobart|tasmania|australia` — added _(2026-05-12)_ - `locations.novartis investigative site|clayton|victoria|australia` — added _(2026-05-12)_ - `locations.novartis investigative site|ghent||belgium` — added _(2026-05-12)_ - `locations.novartis investigative site|sofia||bulgaria` — added _(2026-05-12)_ - `locations.novartis investigative site|hefei|anhui|china` — added _(2026-05-12)_ - `locations.novartis investigative site|nanjing|jiangsu|china` — added _(2026-05-12)_ - `locations.novartis investigative site|chengdu|sichuan|china` — added _(2026-05-12)_ - `locations.novartis investigative site|glostrup municipality||denmark` — added _(2026-05-12)_ - `locations.novartis investigative site|berlin||germany` — added _(2026-05-12)_ - `locations.novartis investigative site|debrecen||hungary` — added _(2026-05-12)_ - `locations.novartis investigative site|sabadell|barcelona|spain` — added _(2026-05-12)_ - `locations.novartis investigative site|vigo|pontevedra|spain` — added _(2026-05-12)_ - `locations.novartis investigative site|valencia|valencia|spain` — added _(2026-05-12)_ - `locations.novartis investigative site|barcelona||spain` — added _(2026-05-12)_ - `locations.novartis investigative site|madrid||spain` — added _(2026-05-12)_ - `locations.novartis investigative site|basel||switzerland` — added _(2026-05-12)_ - `locations.novartis investigative site|lausanne||switzerland` — added _(2026-05-12)_ - `locations.novartis investigative site|taichung|taiwan roc|taiwan` — added _(2026-05-12)_ - `locations.novartis investigative site|kaohsiung city||taiwan` — added _(2026-05-12)_ - `locations.novartis investigative site|taichung||taiwan` — added _(2026-05-12)_ - `locations.novartis investigative site|liverpool||united kingdom` — added _(2026-05-12)_ - `locations.novartis investigative site|swindon||united kingdom` — added _(2026-05-12)_ - `locations.novartis investigative site|tyne and wear||united kingdom` — added _(2026-05-12)_ --- *Canonical: https://parkinsonspathways.com/agent/trials/NCT04035668.md* *Source data (authoritative): https://clinicaltrials.gov/study/NCT04035668* *This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*