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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04035668




Registration number
NCT04035668
Ethics application status
Date submitted
8/07/2019
Date registered
29/07/2019

Titles & IDs
Public title
A Phase 2 Study to Evaluate the Safety and Efficacy of LOU064 in Patients With Moderate to Severe Sjögren's Syndrome
Scientific 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)
Secondary ID [1] 0 0
2018-004387-54
Secondary ID [2] 0 0
CLOU064E12201
Universal Trial Number (UTN)
Trial acronym
LOUiSSe
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Sjögren Syndrome 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Rheumatoid arthritis
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Remibrutinib
Treatment: Drugs - Placebo

Experimental: Remibrutinib 100 mg bid - Remibrutinib 100 mg twice daily (bid)

Experimental: Remibrutinib 100 mg qd - Remibrutinib 100 mg once daily (qd)

Placebo comparator: Placebo - Placebo group


Treatment: Drugs: Remibrutinib
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.

Treatment: Drugs: Placebo
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.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) Total Score at Week 24
Timepoint [1] 0 0
Baseline, Week 24
Secondary outcome [1] 0 0
Change From Baseline in ESSDAI Total Score Over Time
Timepoint [1] 0 0
Baseline, Week 2, Week 4, Week 8, Week 12, Week 16 and Week 20
Secondary outcome [2] 0 0
Change From Baseline in EULAR Sjögren's Syndrome Patient Reported Index (ESSPRI) Total Score Over Time
Timepoint [2] 0 0
Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24
Secondary outcome [3] 0 0
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-F) Total Score Over Time
Timepoint [3] 0 0
Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24
Secondary outcome [4] 0 0
Change From Baseline in EuroQual 5 Dimensions (EQ-5D) VAS Score Over Time
Timepoint [4] 0 0
Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24
Secondary outcome [5] 0 0
Change From Baseline in Physician Global Assessment Scale (PhGA) Score Over Time
Timepoint [5] 0 0
Baseline, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24
Secondary outcome [6] 0 0
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Timepoint [6] 0 0
From first dose of study treatment up 30 days after last dose (Week 29)
Secondary outcome [7] 0 0
Maximum Observed Blood Concentration (Cmax) of Remibrutinib at Week 4
Timepoint [7] 0 0
pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4
Secondary outcome [8] 0 0
Maximum Observed Blood Concentration (Cmax) of Remibrutinib at Week 24
Timepoint [8] 0 0
pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 24
Secondary outcome [9] 0 0
Time to Reach Maximum Observed Blood Concentration (Tmax) of Remibrutinib at Week 4
Timepoint [9] 0 0
pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4
Secondary outcome [10] 0 0
Time to Reach Maximum Observed Blood Concentration (Tmax) of Remibrutinib at Week 24
Timepoint [10] 0 0
pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 24
Secondary outcome [11] 0 0
Area Under the Blood Concentration-time Curve Within a Dosing Interval (Tau) at Steady-state (AUCtau) of Remibrutinib at Week 4
Timepoint [11] 0 0
pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4
Secondary outcome [12] 0 0
Area Under the Blood Concentration-time Curve Within a Dosing Interval (Tau) at Steady-state (AUCtau) of Remibrutinib at Week 24
Timepoint [12] 0 0
pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 24
Secondary outcome [13] 0 0
Area Under the Blood Concentration-time Curve From Time Zero to 4 Hours Post-dose (AUC0-4h) of Remibrutinib at Week 4
Timepoint [13] 0 0
pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4
Secondary outcome [14] 0 0
Area Under the Blood Concentration-time Curve From Time Zero to 4 Hours Post-dose (AUC0-4h) of Remibrutinib at Week 24
Timepoint [14] 0 0
pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 24
Secondary outcome [15] 0 0
Elimination Half-life (T1/2) of Remibrutinib at Week 4
Timepoint [15] 0 0
pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 4
Secondary outcome [16] 0 0
Elimination Half-life (T1/2) of Remibrutinib at Week 24
Timepoint [16] 0 0
pre-dose, 0.5, 1, 2, 3 and 4 hours post-dose at Week 24

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Other
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s

The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA,TAS,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Woodville
Recruitment hospital [2] 0 0
Novartis Investigative Site - Hobart
Recruitment hospital [3] 0 0
Novartis Investigative Site - Clayton
Recruitment postcode(s) [1] 0 0
5011 - Woodville
Recruitment postcode(s) [2] 0 0
7000 - Hobart
Recruitment postcode(s) [3] 0 0
3168 - Clayton
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Massachusetts
Country [2] 0 0
Belgium
State/province [2] 0 0
Gent
Country [3] 0 0
Bulgaria
State/province [3] 0 0
Sofia
Country [4] 0 0
China
State/province [4] 0 0
Anhui
Country [5] 0 0
China
State/province [5] 0 0
Jiangsu
Country [6] 0 0
China
State/province [6] 0 0
Sichuan
Country [7] 0 0
Denmark
State/province [7] 0 0
Glostrup
Country [8] 0 0
Germany
State/province [8] 0 0
Berlin
Country [9] 0 0
Hungary
State/province [9] 0 0
Debrecen
Country [10] 0 0
Spain
State/province [10] 0 0
Barcelona
Country [11] 0 0
Spain
State/province [11] 0 0
Comunidad Valenciana
Country [12] 0 0
Spain
State/province [12] 0 0
Pontevedra
Country [13] 0 0
Spain
State/province [13] 0 0
Madrid
Country [14] 0 0
Switzerland
State/province [14] 0 0
Basel
Country [15] 0 0
Switzerland
State/province [15] 0 0
Lausanne
Country [16] 0 0
Taiwan
State/province [16] 0 0
Taiwan ROC
Country [17] 0 0
Taiwan
State/province [17] 0 0
Kaohsiung
Country [18] 0 0
Taiwan
State/province [18] 0 0
Taichung
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Liverpool
Country [20] 0 0
United Kingdom
State/province [20] 0 0
Swindon
Country [21] 0 0
United Kingdom
State/province [21] 0 0
Tyne And Wear

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.