A Single Dose, Double-blind, Placebo-controlled, Parallel Study to Assess the Pharmacodynamics, Pharmacokinetics and Safety and Tolerability of VAY736 in Patients With Primary Sjögren's Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 27
- 试验地点
- 1
- 主要终点
- Overall Incidence of Adverse Events
研究概览
简要总结
This study was designed to evaluate the safety, tolerability, pharmacokinetics and therapeutic efficacy of a single intravenous infusion of VAY7346 monoclonal antibody in pSS patients
详细描述
Patients were enrolled in 2 sequential cohorts:
Cohort 1: 6 patients received 3 mg/kg or Placebo (2:1 ratio) Cohort 2: 21 patients received 10 mg/kg, 3 mg/kg or Placebo (6:1:3 ratio)
At week 24 the blind was broken to assess continuation in the trial:
- If a patient received VAY736 and their B cell recovery was demonstrated at Week 24, then patients completed the trial.
- If a patient received VAY736 and their B cell recovery was NOT demonstrated at Week 24, then patients were followed up until B cell recovery was demonstrated
- If a patient received placebo, they were offered the option of receiving open-label VAY736 (10 mg/kg) in a separate treatment arm.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Treatment was unblinded at an individual patient level at Week 24 to determine their progress in the study (follow-up, open-label VAY736 or End of Study Visit).
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Placebo
single dose iv of Placebo. At Week 24 patients were offered to receive open label VAY736 10 mg/kg.
干预措施: Placebo (Drug)
VAY736 10 mg/kg
single dose iv of VAY736 at a dose of 10mg/kg
干预措施: VAY736 (Drug)
VAY736 3 mg/kg
single dose iv of VAY736 at a dose of 3mg/kg
干预措施: VAY736 (Drug)
结局指标
主要结局
Overall Incidence of Adverse Events
时间窗: Baseline to Week 24
Number of subjects with Adverse Events during the double blind treatment period.
Change in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI)
时间窗: Baseline, week 12
The effect of VAY736 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.
次要结局
- Change in Short Form (36) Health Survey (SF-36)(Baseline, week 12)
- VAY736 Serum Concentration - AUCinf(0, 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks.)
- VAY736 Serum Concentration - Tmax(0, 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks.)
- Change in Multidimensional Fatigue Inventory (MFI)(Baseline, week 12)
- VAY736 Serum Concentration - CL(0, 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks.)
- Change in EULAR Sjögren's Syndrome Patient Response Index (ESSPRI)(Baseline, week 12)
- Change in the Physician's Global Assessment of Overall Disease Activity by Means of Visual Analog Scale (VAS)(Baseline, week 12)
- Change in the Patient's Global Assessment of Overall Disease Activity by Means of Visual Analog Scale (VAS)(Baseline, week 12)
- VAY736 Serum Concentration - Vz(0, 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks.)
- VAY736 Serum Concentration - AUClast(0, 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks.)
- VAY736 Serum Concentration - Cmax(0, 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks.)
- VAY736 Serum Concentration - T1/2(0, 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks.)