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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03905525




Registration number
NCT03905525
Ethics application status
Date submitted
25/03/2019
Date registered
5/04/2019

Titles & IDs
Public title
Study of Safety and Efficacy of Multiple Doses of CFZ533 in Two Distinct Populations of Patients With Sjogren's Syndrome
Scientific title
A 48-week, 6-arm, Randomized, Double-blind, Placebo-controlled Multicenter Trial to Assess the Safety and Efficacy of Multiple CFZ533 Doses Administered Subcutaneously in Two Distinct Populations of Patients With Sjogren's Syndrome (TWINSS)
Secondary ID [1] 0 0
2018-004476-35
Secondary ID [2] 0 0
CCFZ533B2201
Universal Trial Number (UTN)
Trial acronym
TWINSS
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 - CFZ533
Other interventions - Placebo

Experimental: Cohort 1 /Arm A - CFZ533 dose 1

Experimental: Cohort 1/Arm B - CFZ533 dose 2

Experimental: Cohort 1/Arm C - CFZ533 dose 3

Placebo comparator: Cohort 1/Arm D - Placebo dose (up to week 24)

Experimental: Cohort 1/Arm D1 - CFZ533 dose 1 (from week 24)

Experimental: Cohort 2/Arm E - CFZ533 dose 1

Placebo comparator: Cohort 2/Arm F - Placebo dose (up to week 24)

Experimental: Cohort 2/Arm F1 - CFZ533 dose 2 (from week 24)


Treatment: Drugs: CFZ533
Biological

Other interventions: Placebo
liquid placebo for injections
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in EULAR Sjögren Syndrome Disease Activity Index (ESSDAI) score from baseline at 24 weeks as compared to placebo
Timepoint [1] 0 0
24 weeks
Primary outcome [2] 0 0
Change in EULAR Sjögren Syndrome Patient Reported Index (ESSPRI) score from baseline at 24 weeks as compared to placebo.
Timepoint [2] 0 0
24 weeks
Secondary outcome [1] 0 0
Change from baseline in ESSPRI at Week 24
Timepoint [1] 0 0
24 weeks
Secondary outcome [2] 0 0
Change from baseline in score of Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) questionnaire at Week 24
Timepoint [2] 0 0
24 weeks
Secondary outcome [3] 0 0
Change from baseline in Physician Global Assessment (PhGA) at Week 24
Timepoint [3] 0 0
24 weeks
Secondary outcome [4] 0 0
Change from baseline in ESSDAI at Week 24
Timepoint [4] 0 0
24 weeks
Secondary outcome [5] 0 0
Proportion of subjects with at least 12 points improvement measured by score of Impact of Dry Eye on Everyday Life (IDEEL) questionnaire symptom bother module at Week 24.
Timepoint [5] 0 0
24 weeks
Secondary outcome [6] 0 0
Incidence of adverse events (AEs), serious adverse events (SAEs) from baseline to Week 24 and from week 24 to the end of study
Timepoint [6] 0 0
60 weeks
Secondary outcome [7] 0 0
Serum Free Light Chain (FLC) levels at analysis visit up to end of study
Timepoint [7] 0 0
60 weeks
Secondary outcome [8] 0 0
Immunoglobulin IgG and IgM levels at analysis visits up to end of study
Timepoint [8] 0 0
60 weeks
Secondary outcome [9] 0 0
Percent change from baseline in plasma CXCL-13 levels at analysis visits up to end of study
Timepoint [9] 0 0
60 weeks

Eligibility
Key inclusion criteria
* Signed informed consent
* Male or female patient = 18 years of age
* Classification of Sjögren's Syndrome according to ACR/EULAR 2016 criteria (Shiboski et al 2017)
* Seropositive for anti-Ro/SSA antibodies
* Stimulated whole salivary flow rate of = 0.1 mL/min

Inclusion criteria specific for Cohort 1:

* ESSDAI = 5 within the 8 predefined organ domains
* ESSPRI score of =5

Inclusion criteria specific for Cohort 2:

* ESSDAI < 5 within 8 domains scored for inclusion criterion for Cohort 1
* ESSPRI fatigue subscore = 5 or ESSPRI dryness subscore = 5
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Sjögren's Syndrome overlap syndromes where another autoimmune rheumatic disease constitutes the principle illness
* Use of other investigational drugs
* Prior use of B cell depleting therapies, abatacept or any other immunosuppressants unless specifically allowed be the protocol.
* Use of steroids at dose >10 mg/day.
* Uncontrolled ocular rosacea (affecting the eye adnexa), posterior blepharitis or Meibomian gland disease (this criterion applies only to patients considered for Cohort 2)
* Active viral, bacterial or other infections requiring systemic treatment
* Receipt of live/attenuated vaccine within a 2-month period prior to randomization.
* Chronic infection with hepatitis B (HBV) or hepatitis C (HCV).
* Evidence of active tuberculosis (TB) infection.

Study design
Purpose of the study
Treatment
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 administering 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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/10/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
6/06/2023
Sample size
Target
Accrual to date
Final
273
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
Novartis Investigative Site - Nedlands
Recruitment postcode(s) [1] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Georgia
Country [2] 0 0
United States of America
State/province [2] 0 0
Indiana
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United States of America
State/province [3] 0 0
Louisiana
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
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United States of America
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New York
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United States of America
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Pennsylvania
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United States of America
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Wisconsin
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Argentina
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Buenos Aires
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Argentina
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Caba
Country [11] 0 0
Austria
State/province [11] 0 0
Graz
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Austria
State/province [12] 0 0
Wien
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Brazil
State/province [13] 0 0
ES
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Brazil
State/province [14] 0 0
MG
Country [15] 0 0
Brazil
State/province [15] 0 0
SP
Country [16] 0 0
Canada
State/province [16] 0 0
Ontario
Country [17] 0 0
Canada
State/province [17] 0 0
Quebec
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Chile
State/province [18] 0 0
Los Rios
Country [19] 0 0
Chile
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RM
Country [20] 0 0
Chile
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Concepcion
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Chile
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Santiago
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Colombia
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Antioquia
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Colombia
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Atlantico
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Colombia
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Valle Del Cauca
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France
State/province [25] 0 0
Brest
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France
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Le Kremlin Bicetre
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France
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Lille
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France
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Paris
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France
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Strasbourg
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Germany
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Bonn
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Germany
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Dresden
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Germany
State/province [32] 0 0
Freiburg
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Germany
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Wuerzburg
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Greece
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Athens
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Hungary
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Fejer
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Hungary
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Budapest
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Hungary
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Szeged
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Israel
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Haifa
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Israel
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Kfar Saba
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Israel
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Ramat Gan
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Italy
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MI
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Italy
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PI
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Italy
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UD
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Japan
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Aichi
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Japan
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Nagasaki
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Japan
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Okayama
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Japan
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Tokyo
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Korea, Republic of
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Seocho Gu
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Netherlands
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Groningen
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Netherlands
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Rotterdam
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Portugal
State/province [51] 0 0
Almada
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Portugal
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Lisboa
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Portugal
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Ponte de Lima
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Romania
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Brasov
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Romania
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Cluj Napoca
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Russian Federation
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Ekaterinburg
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Russian Federation
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Kazan
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Russian Federation
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Moscow
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Russian Federation
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Orenburg
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Russian Federation
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St Petersburg
Country [61] 0 0
Russian Federation
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Tomsk
Country [62] 0 0
Sweden
State/province [62] 0 0
SE
Country [63] 0 0
Turkey
State/province [63] 0 0
Ankara
Country [64] 0 0
United Kingdom
State/province [64] 0 0
Birmingham
Country [65] 0 0
United Kingdom
State/province [65] 0 0
Doncaster
Country [66] 0 0
United Kingdom
State/province [66] 0 0
Manchester

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
Study Director 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 access to patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to protect 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?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03905525