A Phase 1b, Open-label, Single-arm Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of JTE-162 in Subjects With Cryopyrin-Associated Periodic Syndrome (CAPS)
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Change and percent change from baseline to end of treatment (EOT) in high-sensitivity C-reactive protein (hs-CRP)
研究概览
简要总结
This study will evaluate the efficacy, safety, tolerability and pharmacokinetics of JTE-162 administered once daily for 2 weeks in subjects with cryopyrin-associated periodic syndrome (CAPS)
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with familial cold autoinflammatory syndrome (FCAS) or Muckle-Wells syndrome (MWS) confirmed by:
- •Clinical History: At least 2 typical clinical symptoms (e.g., urticarial skin rash, myalgia, arthralgia, recurrent fever, fatigue/malaise, conjunctivitis or other autoinflammatory symptoms) prior to the Screening Visit; AND
- •Genetic Confirmation: Confirmed nucleotide-binding and oligomerization domain (NOD)-like receptor family pyrin domain containing 3 (NLRP3) mutation;
- •Willing to discontinue current anti-interleukin (IL)-1 treatment, if applicable;
- •Demonstrates de novo flaring of CAPS during the Screening Period.
排除标准
- •Has chronic infantile neurologic cutaneous articular syndrome (CINCA)/neonatal-onset multisystem inflammatory disease (NOMID);
- •Has a history or presence of amyloidosis, progressive hearing loss, organ damage or any symptom contraindicating anti-IL-1 treatment washout;
- •Has active systemic bacterial, fungal or viral infection(s) within 14 days prior to Day 1 or a history of clinically significant recurrent infectious diseases
研究组 & 干预措施
JTE-162 Tablets
Dose 1 once daily for 2 Weeks
干预措施: JTE-162 (Drug)
结局指标
主要结局
Change and percent change from baseline to end of treatment (EOT) in high-sensitivity C-reactive protein (hs-CRP)
时间窗: 2 Weeks
Change and percent change from baseline to EOT in Serum amyloid A (SAA)
时间窗: 2 Weeks
Change and percent change from baseline to EOT in Interleukin (IL)-6
时间窗: 2 Weeks
Change and percent change from baseline to EOT in Key symptom score (KSS)
时间窗: 2 Weeks
Change and percent change from baseline to EOT in Individual symptom score on Daily Health Assessment Form, Second Generation (DHAF2)
时间窗: 2 Weeks
Change and percent change from baseline to EOT in Global assessments of disease activity on DHAF2
时间窗: 2 Weeks
Change and percent change from baseline to EOT in Global CAPS symptom assessment by the Investigator
时间窗: 2 Weeks
Change and percent change from baseline to EOT in Individual CAPS symptom assessment by the Investigator
时间窗: 2 Weeks
Number of adverse events
时间窗: 4 Weeks
JTE-162 post-dose plasma concentrations on Day 1
时间窗: 1 Day
JTE-162 trough plasma concentrations on Days 2, 3 and 4, and at the Week 2 Visit (Day 15±2)
时间窗: Day 2, Day 3, Day 4 and Day 15±2
次要结局
未报告次要终点