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
Overview
- Phase
- Phase 2
- Intervention
- Placebo
- Conditions
- Primary Sjögren's Syndrome
- Sponsor
- Novartis Pharmaceuticals
- Enrollment
- 30
- Locations
- 1
- Primary Endpoint
- Number of Participants With Primary Sjögren's Syndrome With Adverse Events and Death up to Day 85
- Status
- Completed
- Last Updated
- 5 years ago
Overview
Brief 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.
Detailed 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.
Investigators
Eligibility Criteria
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.
Arms & Interventions
Placebo
Capsule matching Placebo
Intervention: Placebo
CDZ173
Capsule
Intervention: CDZ173
Outcomes
Primary Outcomes
Number of Participants With Primary Sjögren's Syndrome With Adverse Events and Death up to Day 85
Time Frame: up to Day 85
Safety and tolerability of CDZ173 in patients with primary Sjögren's syndrome up to End of Treatment Day 85
Change From Baseline in the EULAR Sjögren's Syndrome Patient Reported Intensity (ESSPRI) After 12 Weeks of Treatment Day 85
Time Frame: Baseline and 12 weeks (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.
Secondary Outcomes
- Change From Baseline in the EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) After 12 Weeks of Treatment Day 85(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(Baseline and 12 weeks (Day 85))
- Change in Baseline in Multidimensional Fatigue Inventory (MFI) After 12 Weeks of Treatment (Day 85)(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(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(Baseline and 12 weeks)