Trial · NCT04035668
A Phase 2 Study to Evaluate the Safety and Efficacy of LOU064 in Patients With Moderate to Severe Sjögren's Syndrome
NCT04035668, Phase 2, Terminated, Novartis Pharmaceuticals; results first posted 2022-12-20
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Phase 2 Study to Evaluate the Safety and Efficacy of LOU064 in Patients With Moderate to Severe Sjögren's Syndrome. Trials, record NCT04035668. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04035668
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 2 Study to Evaluate the Safety and Efficacy of LOU064 in Patients With Moderate to Severe Sjögren's Syndrome
- NCT number
- NCT04035668
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 73 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Sjögren Syndrome
- Interventions
- Remibrutinib, Placebo
- First posted
- 2019-07-29
- Last updated
- 2023-01-30
- Other registries' IDs
- eudract:2018-004387-54
- Results first posted
- 2022-12-20
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) Total Score at Week 24 | 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 | Change From Baseline in ESSDAI Total Score Over Time | Baseline, Week 2, Week 4, Week 8, Week 12, Week 16 and Week 20 | 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 | Change From Baseline in EULAR Sjögren's Syndrome Patient Reported Index (ESSPRI) Total Score Over Time | Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24 | ESSPRI is an established disease outcome measure for Sjögren's Syndrome. It consists of three domains of dryness, pain, and fatigue. The patient can assess the severity of symptoms they experience on a single 0-10 numerical scale for each of the three domains. The ESSPRI score is defined as the mean of scores from the three scales: (dryness + pain +fatigue) /3. ESSPRI total score ranges from 0 to 10 with higher values indicating more disease symptoms. 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 ESSPRI for all post-baseline time points up to Week 24. Values estimated from the model are presented in this table. |
| Secondary | Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-F) Total Score Over Time | Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24 | FACIT-F v4 is a short, 13-item, easy-to-administer tool that measures an individual's level of fatigue during their usual daily activities over the past week. The level of fatigue was measured on a 5-point Likert scale (0 = not at all, 1 = a little bit, 2 = somewhat, 3 = quite a bit, 4 = very much). FACIT-F total score ranges from 0 to 52 with higher values indicating higher quality of life (less fatigue). A positive change from baseline is a favorable outcome. 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 FACIT-F for all post-baseline time points up to Week 24. Values estimated from the model are presented in this table. |
| Secondary | Change From Baseline in EuroQual 5 Dimensions (EQ-5D) VAS Score Over Time | Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24 | EQ-5D is a standardized instrument that measures the health-related quality of life. The EQ-5D consists of a descriptive system and a visual analog scale (VAS).The EQ-5D VAS records the patient's self-rated health on a vertical visual analogue scale with 0 representing 'Worst imaginable Health State' and 100 'Best imaginable Health State'. A positive change from baseline is a favorable outcome. 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 EQ-5D VAS score for all post-baseline time points up to Week 24. Values estimated from the model are presented in this table. |
| Secondary | Change From Baseline in Physician Global Assessment Scale (PhGA) Score Over Time | Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24 | The physician's global assessment scale was used for the Investigator to rate the disease activity of their patient using 100 mm visual analog scale (VAS) ranging from "no disease activity" (0) to "maximal disease activity" (100). 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 PhGA score for all post-baseline time points up to Week 24. Values estimated from the model are presented in this table. |
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | From first dose of study treatment up 30 days after last dose (Week 29) | Number of participants with TEAEs and serious TEAEs, including changes from baseline in vital signs, electrocardiograms and laboratory results qualifying and reported as TEAEs. TEAEs are defined as adverse events that started after the first dose of study medications or adverse events present prior to start of double-blind treatment but increased in severity. The number of participants in each category is reported in the table. |
| Secondary | Maximum Observed Blood Concentration (Cmax) of Remibrutinib at Week 4 | pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4 | Remibrutinib was determined in whole blood by a validated liquid chromatography-tandem mass spectrometry (LC-MS/MS) method with a lower limit of quantification (LLOQ) of 1.0 ng/mL. Pharmacokinetic (PK) parameters were calculated based on remibrutinib blood concentrations by using the actual recorded sampling times and non-compartmental methods with Phoenix WinNonlin (version 8 or higher). Concentrations below the LLOQ were treated as zero. Cmax is defined as the maximum (peak) observed blood concentration following a dose. |
| Secondary | Maximum Observed Blood Concentration (Cmax) of Remibrutinib at Week 24 | pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 24 | Remibrutinib was determined in whole blood by a validated liquid chromatography-tandem mass spectrometry (LC-MS/MS) method with a lower limit of quantification (LLOQ) of 1.0 ng/mL. Pharmacokinetic (PK) parameters were calculated based on remibrutinib blood concentrations by using the actual recorded sampling times and non-compartmental methods with Phoenix WinNonlin (version 8 or higher). Concentrations below the LLOQ were treated as zero. Cmax is defined as the maximum (peak) observed blood concentration following a dose. |
| Secondary | Time to Reach Maximum Observed Blood Concentration (Tmax) of Remibrutinib at Week 4 | pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4 | Remibrutinib was determined in whole blood by a validated LC-MS/MS method with a LLOQ of 1.0 ng/mL. Pharmacokinetic (PK) parameters were calculated based on remibrutinib blood concentrations by using the actual recorded sampling times and non-compartmental methods with Phoenix WinNonlin (version 8 or higher). Concentrations below the LLOQ were treated as zero. Tmax is defined as the time to reach maximum (peak) blood concentration following a dose. |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) Total Score at Week 24 | Baseline, Week 24 | Participants in the Full Analysis Set (FAS) with a valid assessment for the outcome measure. FAS included all participants who were randomized in the study. | score on scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in ESSDAI Total Score Over Time | Baseline, Week 2, Week 4, Week 8, Week 12, Week 16 and Week 20 | Participants in the Full Analysis Set (FAS) with a valid assessment for the outcome measure. FAS included all participants who were randomized in the study. | score on scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in EULAR Sjögren's Syndrome Patient Reported Index (ESSPRI) Total Score Over Time | Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24 | Participants in the Full Analysis Set (FAS) with a valid assessment for the outcome measure. FAS included all participants who were randomized in the study. | score on scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-F) Total Score Over Time | Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24 | Participants in the Full Analysis Set (FAS) with a valid assessment for the outcome measure. FAS included all participants who were randomized in the study. | score on scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in EuroQual 5 Dimensions (EQ-5D) VAS Score Over Time | Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24 | Participants in the Full Analysis Set (FAS) with a valid assessment for the outcome measure. FAS included all participants who were randomized in the study. | score on scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Physician Global Assessment Scale (PhGA) Score Over Time | Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24 | Participants in the Full Analysis Set (FAS) with a valid assessment for the outcome measure. FAS included all participants who were randomized in the study. | score on scale | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | From first dose of study treatment up 30 days after last dose (Week 29) | Safety Analysis Set defined as participants who received at least one dose of the study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Maximum Observed Blood Concentration (Cmax) of Remibrutinib at Week 4 | pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4 | Participants in the PK Analysis Set (PKS) with a valid value for the outcome measure. PKS is defined as participants with at least one available valid PK concentration measurement, who received any study drug, and with no protocol deviations that impact PK data. | ng/mL | MEAN |
| SECONDARY | Maximum Observed Blood Concentration (Cmax) of Remibrutinib at Week 24 | pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 24 | Participants in the PK Analysis Set (PKS) with a valid value for the outcome measure. PKS is defined as participants with at least one available valid PK concentration measurement, who received any study drug, and with no protocol deviations that impact PK data. | ng/mL | MEAN |
| SECONDARY | Time to Reach Maximum Observed Blood Concentration (Tmax) of Remibrutinib at Week 4 | pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4 | Participants in the PK Analysis Set (PKS) with a valid value for the outcome measure. PKS is defined as participants with at least one available valid PK concentration measurement, who received any study drug, and with no protocol deviations that impact PK data. | hours | MEDIAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group169
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) Total Score at Week 24 | Remibrutinib 100 mg qd | -4.70 | Standard Error 0.78 | score on scale | |
| Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) Total Score at Week 24 | Remibrutinib 100 mg Bid | -3.70 | Standard Error 0.80 | score on scale | |
| Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) Total Score at Week 24 | Any Remibrutinib | -4.20 | Standard Error 0.56 | score on scale | |
| Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) Total Score at Week 24 | Placebo | -1.34 | Standard Error 0.74 | score on scale | |
| Change From Baseline in ESSDAI Total Score Over Time | Remibrutinib 100 mg qd | Week 2 | -1.68 | Standard Error 0.52 | score on scale |
| Change From Baseline in ESSDAI Total Score Over Time | Remibrutinib 100 mg Bid | Week 2 | -1.05 | Standard Error 0.52 | score on scale |
| Change From Baseline in ESSDAI Total Score Over Time | Any Remibrutinib | Week 2 | -1.37 | Standard Error 0.37 | score on scale |
| Change From Baseline in ESSDAI Total Score Over Time | Placebo | Week 2 | -0.37 | Standard Error 0.55 | score on scale |
| Change From Baseline in ESSDAI Total Score Over Time | Remibrutinib 100 mg qd | Week 4 | -2.57 | Standard Error 0.60 | score on scale |
| Change From Baseline in ESSDAI Total Score Over Time | Remibrutinib 100 mg Bid | Week 4 | -2.54 | Standard Error 0.62 | score on scale |
1 to 10 of 169Source ClinicalTrials.gov
See all 169 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Remibrutinib 100 mg Bid | 24 | |
| Overall Study | STARTED | Remibrutinib 100 mg qd | 25 | |
| Overall Study | STARTED | Placebo | 24 | |
| Overall Study | COMPLETED | Remibrutinib 100 mg Bid | 17 | |
| Overall Study | COMPLETED | Remibrutinib 100 mg qd | 17 | |
| Overall Study | COMPLETED | Placebo | 21 | |
| Overall Study | NOT COMPLETED | Remibrutinib 100 mg Bid | 7 | |
| Overall Study | NOT COMPLETED | Remibrutinib 100 mg qd | 8 | |
| Overall Study | NOT COMPLETED | Placebo | 3 |
Source ClinicalTrials.gov
Adverse events135
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Leukopenia | Blood and lymphatic system disorders | Remibrutinib 100 mg Bid | 0 | 24 | |
| other | Leukopenia | Blood and lymphatic system disorders | Remibrutinib 100 mg qd | 0 | 25 | |
| other | Leukopenia | Blood and lymphatic system disorders | Any Remibrutinib | 0 | 49 | |
| other | Leukopenia | Blood and lymphatic system disorders | Placebo | 3 | 24 | |
| other | Leukopenia | Blood and lymphatic system disorders | Total | 3 | 73 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Remibrutinib 100 mg Bid | 0 | 24 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Remibrutinib 100 mg qd | 0 | 25 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Any Remibrutinib | 0 | 49 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Placebo | 3 | 24 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Total | 3 | 73 |
1 to 10 of 135Source ClinicalTrials.gov
See all 135 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Remibrutinib 100 mg Bid | 0 | 24 |
| Total, all-cause mortality | Remibrutinib 100 mg qd | 0 | 25 |
| Total, all-cause mortality | Any Remibrutinib | 0 | 49 |
| Total, all-cause mortality | Placebo | 0 | 24 |
| Total, all-cause mortality | Total | 0 | 73 |
| Total, other adverse events | Remibrutinib 100 mg Bid | 15 | 24 |
| Total, other adverse events | Remibrutinib 100 mg qd | 12 | 25 |
| Total, other adverse events | Any Remibrutinib | 27 | 49 |
| Total, other adverse events | Placebo | 17 | 24 |
| Total, other adverse events | Total | 44 | 73 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37932009 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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