Safety, Pharmacokinetics and Preliminary Efficacy Study of CFZ533 in Patients With Primary Sjögren's Syndrome

Phase 2

Completed

• Conditions: Primary Sjögren's Syndrome
• Interventions: Drug: CFZ533 active - Cohort 2; Drug: CFZ533 placebo - Cohort 2; Drug: CFZ533 active - Cohort 1; Drug: CFZ533 placebo- Cohort 1; Drug: CFZ533 active -Cohort 3; Drug: CFZ533 active - Cohort 3

• Registration Number: NCT02291029
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

This study did evaluate the safety,tolerability and preliminary therapeutic efficacy of multiple doses of intravenous infusion of CFZ533 monoclonal antibody in patients with primary Sjögren's syndrome(pSS)

Detailed Description

Not available

Study Timeline

• Study Start: 2014-10-22
• Study First Submitted: 2014-11-04
• Study First Submitted QC: 2014-11-11
• Study First Posted: 2014-11-14
• Primary Completion: 2018-06-29
• Study Completion: 2018-06-29
• Results First Submitted: 2019-06-28
• Status Verified: 2019-07
• Results First Submitted QC: 2019-07-26
• Results First Posted: 2019-08-14
• Last Update Submitted: 2020-12-09
• Last Update Posted: 2021-01-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 69

Inclusion Criteria

• Diagnosis of primary Sjögren's syndrome
• ESSDAI score ≥ 6

Exclusion Criteria

• Secondary Sjögren's syndrome
• Receiving cyclosphosphamide, corticosteroid bolus with dose over 1 mg/kg, rituximab, belimunab, other immunosuppressives.
• At significant risk for thromboembolic event
• Clinically significant systemic infection
• Significant elevated risk for infection

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
CFZ533 active- Cohort 2	CFZ533 active - Cohort 2	multiple doses of CFZ533 intravenous infusion
CFZ533 placebo- Cohort 2	CFZ533 placebo - Cohort 2	multiple doses of placebo intravenous infusion
CFZ533 active - Cohort 1	CFZ533 active - Cohort 1	multiple doses of CFZ533 s.c. injection
CFZ533 placebo - Cohort 1	CFZ533 placebo- Cohort 1	multiple doses of placebo s.c. injection
CFZ533 Treatment Arm 1 - Cohort 3	CFZ533 active -Cohort 3	multiple doses of CFZ533 s.c. injection
CFZ533 Treatment Arm 2 - Cohort 3	CFZ533 active - Cohort 3	Single dose of CFZ533 i.v. infusion and multiple doses of CFZ533 s.c. injection

Primary Outcome Measures

Name	Time	Method
Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI)	Baseline and Week 12	The effect of CFZ533 on clinical disease activity was measured by the change in ESSDAI (EULAR Sjögren's syndrome disease activity index) between baseline and week 12. The instrument contains 12 organ-specific domains contributing to disease activity. For each domain, features of disease activity are scored in 3 or 4 levels according to their severity.; These scores are then summed across the 12 domains in a weighted manner to provide the total score (range 0-123). A reduction from baseline indicates improvement in patients.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in EULAR Sjögren's Syndrome Patient Reported Intensity (ESSPRI)	Baseline and Week 12	The ESSPRI is a patient self-reported outcome measure to assess dryness, limb pain, fatigue and mental fatigue, where each of the domains normally reported as 0 (not at all) to 10 (extremely severe). The final ESSPRI score is the average of three: dryness, pain and fatigue. A reduction from baseline indicates the improvement of symptoms.
Change From Baseline in Physician Global Assessment of the Patient's Overall Disease Activity (VAS)	Baseline and Week 12	The visual analogue scale used is a 100 mm VAS ranging from "no disease" (0 mm) to "maximal disease activity" (100 mm).
Change From Baseline in Patient's Global Assessment of Their Disease Activity (VAS)	Baseline and Week 12	The visual analogue scale used is a 100 mm VAS ranging from "no disease" (0 mm) to "maximal disease activity" (100 mm).
Change From Baseline in Short Form (36) Health Survey (SF-36) Physical Component Score	Baseline and Week 12	The SF-36 is a 36-item, patient self-reported outcome measure (questionnaires) of patient health. The outcome of the questionnaires in eight scales results in two summary scores, physical component and mental component, both ranging from 0 - 100. An increase from baseline in either component summary score indicates reduced disease burden.
Change From Baseline in Short Form (36) Health Survey (SF-36) Mental Component Score	Baseline and Week 12	The SF-36 is a 36-item, patient self-reported outcome measure (questionnaires) of patient health. The outcome of the questionnaires in eight scales results in two summary scores, physical component and mental component, both ranging from 0 - 100. An increase from baseline in either component summary score indicates reduced disease burden.
Change From Baseline in Multidimensional Fatigue Inventory (MFI)	Baseline and Week 12	The MFI is a patient self-reported outcome measure (questionnaires) to assess fatigue covering the following dimensions: General Fatigue, Physical Fatigue, Mental Fatigue, Reduced Motivation and Reduced Activity. Each dimension has a possible range from 4-20. The reported total score has a range from 20-100. A reduction from baseline in MFI indicates improvement.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇬🇧 Newcastle upon Tyne, United Kingdom