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

Sponsor: Novartis Pharmaceuticals

Sommario: This study will evaluate safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of multiple doses of CFZ533 (iscalimab) in patients with Sjögren's Syndrome.

TIPOLOGIA STUDIO

Interventistico

FASE

Fase 2

TRATTAMENTO

• CFZ533
• Placebo

OBIETTIVO PRIMARIO

• Misura: Change in EULAR Sjögren Syndrome Disease Activity Index (ESSDAI) score from baseline at 24 weeks as compared to placebo
• Tempo: 24 weeks
• Descrizione: Cohort 1 - Efficacy

• Misura: Change in EULAR Sjögren Syndrome Patient Reported Index (ESSPRI) score from baseline at 24 weeks as compared to placebo.
• Tempo: 24 weeks
• Descrizione: Cohort 2 - Efficacy

OBIETTIVO SECONDARIO

• Misura: Change from baseline in ESSPRI at Week 24
• Tempo: 24 weeks
• Descrizione: Cohort 1 - Efficacy (Patient Reported Outcomes)

• Misura: Change from baseline in score of Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) questionnaire at Week 24
• Tempo: 24 weeks
• Descrizione: Cohort 1&2 - Efficacy (Patient Reported Outcomes)

• Misura: Change from baseline in Physician Global Assessment (PhGA) at Week 24
• Tempo: 24 weeks
• Descrizione: Cohort 1&2 - Efficacy (Clinical Outcome Measures)

• Misura: Change from baseline in ESSDAI at Week 24
• Tempo: 24 weeks
• Descrizione: Cohort 2 - Efficacy (Clinical Outcome Measures)

• Misura: 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.
• Tempo: 24 weeks
• Descrizione: Cohort 2 - Efficacy (Patient Reported Outcomes)

• Misura: Incidence of adverse events (AEs), serious adverse events (SAEs) from baseline to Week 24 and from week 24 to the end of study
• Tempo: 60 weeks
• Descrizione: Cohort 1&2 - Safety

• Misura: Serum Free Light Chain (FLC) levels at analysis visit up to end of study
• Tempo: 60 weeks
• Descrizione: Cohort 1&2 - Biomarkers (1)

• Misura: Immunoglobulin IgG and IgM levels at analysis visits up to end of study
• Tempo: 60 weeks
• Descrizione: Cohort 1&2 - Biomarkers (2)

• Misura: Percent change from baseline in plasma CXCL-13 levels at analysis visits up to end of study
• Tempo: 60 weeks
• Descrizione: Cohort 1&2 - Biomarkers (3) Chemokine (C-X-C motif) ligand 13 (CXCL13), also known as B lymphocyte chemoattractant (BLC) or B cell-attracting chemokine 1 (BCA-1), is a protein ligand that in humans is encoded by the CXCL13 gene.

CRITERI DI ELIGIBILITA'

• Criteri di inclusione e esclusione

  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.

SESSO

Tutti

ETA' MINIMA

Età Minima: 18 Years
Età Massima: N/A

LUOGO

Novartis Investigative Site
Milano, 20132

Novartis Investigative Site
Pisa, 56124

Novartis Investigative Site
Udine, 33100