Study of Safety and Efficacy of Multiple Doses of CFZ533 in Two Distinct Populations of Patients With Sjogren's Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 26 September 2023
• Main ID: NCT03905525
• Date of registration: 25/03/2019
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Study of Safety and Efficacy of Multiple Doses of CFZ533 in Two Distinct Populations of Patients With Sjogren's Syndrome TWINSS
• 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)
• Date of first enrolment: October 1, 2019
• Target sample size: 273
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03905525
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Argentina, Australia, Austria, Brazil, Canada, Chile, Colombia, France, Germany, Greece, Hungary, Israel, Italy, Japan, Korea, Republic of, Netherlands, Portugal, Romania, Russian Federation, Slovenia, Sweden, Turkey, United Kingdom, United States

Contacts

• Name: Study Director Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion criteria

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

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Sjögren Syndrome

Intervention(s)

Drug: CFZ533

Other: Placebo

Primary Outcome(s)

Change in EULAR Sjögren Syndrome Disease Activity Index (ESSDAI) score from baseline at 24 weeks as compared to placebo [Time Frame: 24 weeks]

Change in EULAR Sjögren Syndrome Patient Reported Index (ESSPRI) score from baseline at 24 weeks as compared to placebo. [Time Frame: 24 weeks]

Secondary Outcome(s)

Change from baseline in ESSPRI at Week 24 [Time Frame: 24 weeks]

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. [Time Frame: 24 weeks]

Incidence of adverse events (AEs), serious adverse events (SAEs) from baseline to Week 24 and from week 24 to the end of study [Time Frame: 60 weeks]

Change from baseline in ESSDAI at Week 24 [Time Frame: 24 weeks]

Percent change from baseline in plasma CXCL-13 levels at analysis visits up to end of study [Time Frame: 60 weeks]

Change from baseline in Physician Global Assessment (PhGA) at Week 24 [Time Frame: 24 weeks]

Change from baseline in score of Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) questionnaire at Week 24 [Time Frame: 24 weeks]

Serum Free Light Chain (FLC) levels at analysis visit up to end of study [Time Frame: 60 weeks]

Immunoglobulin IgG and IgM levels at analysis visits up to end of study [Time Frame: 60 weeks]

Secondary ID(s)

CCFZ533B2201

2018-004476-35

Source(s) of Monetary Support

Please refer to primary and secondary sponsors