Safety, Pharmacokinetics, and Preliminary Efficacy Study of CDZ173 in Patients With Primary Sjögren's Syndrome

Primary Sjögren's Syndrome Clinical Trial

Official title:

A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CDZ173 in Patients With Primary Sjögren's Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02775916
• Other study ID #: CCDZ173X2203
• Secondary ID: 2014-004616-12
• Status: Completed
• Phase: Phase 2
• Start date: June 1, 2016
• Est. completion date: May 17, 2017

Study information

• Verified date: September 2019
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This Study is designed to evaluate the safety, tolerability, pharmacokinetics and preliminary therapeutic efficacy of oral administrations of CDZ173 in patients with primary Sjögren's syndrome.

Description:

This study is designed to evaluate the safety, tolerability, pharmacokinetics and preliminary therapeutic efficacy of oral administrations of CDZ173, a selective PI3K delta inhibitor, for 12 weeks, in patients with primary Sjögren's syndrome. Data from this study will provide the basis for further development of the compound for the treatment of primary Sjögren's syndrome.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: May 17, 2017
• Est. primary completion date: May 17, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

Inclusion Criteria:

• Diagnosis of primary Sjögren's syndrome (pSS)
• ESSDAI score = 6 at screening visit

Exclusion Criteria:

• Secondary Sjögren's syndrome Other protocol-defined inclusion/exclusion criteria may apply.

Related Conditions & MeSH terms

• Primary Sjögren's Syndrome
• Sjogren's Syndrome
• Syndrome

Intervention

Drug:
• CDZ173

• Placebo

Locations

Country	Name	City	State
Germany	Novartis Investigative Site	Berlin
Hungary	Novartis Investigative Site	Debrecen

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

Germany,  Hungary, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Primary Sjögren's Syndrome With Adverse Events and Death up to Day 85	Safety and tolerability of CDZ173 in patients with primary Sjögren's syndrome up to End of Treatment Day 85	up to Day 85
Primary	Change From Baseline in the EULAR Sjögren's Syndrome Patient Reported Intensity (ESSPRI) After 12 Weeks of Treatment Day 85	The ESSPRI is an established disease outcome measure for Sjögren's syndrome. The ESSPRI is a patient-reported, subjective symptom index for primary Sjögren's syndrome developed by the EULAR consortium. It consists of three questions covering the cardinal symptoms of Sjögren's syndrome: dryness, fatigue and pain (articular and/or muscular). Each domain scored on scale of 0-10 (0 =no symptom at all and 10 = worst symptom imaginable), and an overall score is calculated as the mean of the three individual domains where all domains carry the same weight. Minimum score can be 0 and maximum score can be 10.	Baseline and 12 weeks (Day 85)
Secondary	Change From Baseline in the EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) After 12 Weeks of Treatment Day 85	The ESSDAI is an established disease outcome measure for Sjögren's syndrome. 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. A reduction from baseline (i.e., a negative change from baseline) in the ESSDAI score is indicative of improvement in a patient.). Each domain is assessed for activity level (i.e., no, low, moderate, high) and assigned a numerical score based on pre-determined weighting of each individual domain. An overall score is then calculated as the sum of all individual weighted domain scores. Overall score is calculated as sum of all individual weighted domain scores (ranges from 0 (best) to 123 (worst activity).	Baseline and 12 weeks (Day 85)
Secondary	Change From Baseline in the Short Form (36) Health Survey (SF-36) After 12 Weeks of Treatment Day 85	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.	Baseline and 12 weeks (Day 85)
Secondary	Change in Baseline in Multidimensional Fatigue Inventory (MFI) After 12 Weeks of Treatment (Day 85)	The Multidimensional Fatigue Inventory (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.; The reported total score has a range from 20-100, higher scores are associated with greater fatigue.	Baseline and 12 weeks (Day 85)
Secondary	Change From Baseline in Physician Global Assessment of the Patient's Overall Disease Activity (Physician VAS) After 12 Weeks of Treatment Day 85	A reduction from baseline (i.e., a negative change from baseline) in physician global VAS assessment score indicates improvement in patients. The visual analogue scale used is a 100 mm VAS ranging from "no disease" (0 mm) to "maximal disease activity" (100 mm).	Baseline and 12 weeks (Day 85)
Secondary	Change From Baseline in Patient's Global Assessment of Their Disease Activity (VAS) After 12 Weeks of Treatment Day 85	A reduction from baseline (or, a negative change from baseline) in patient global VAS assessment score indicates improvement in patients.; The visual analogue scale used is a 100 mm VAS ranging from "no disease" (0 mm) to "maximal disease activity" (100 mm).	Baseline and 12 weeks

External Links

•   A Plain Language Trial Summary is available on novartisclinicatrials.com