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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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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

Conditions
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Primary Sjögren's Syndrome

Intervention / Treatment
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* Drug: CDZ173
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CDZ173<br>Capsule | Drug: CDZ173<br> <br> |
| Placebo Comparator: Placebo<br>Capsule matching Placebo | Drug: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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) | 
| 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) | 
| 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) | 
| 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) | 
| 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 |