A Phase 2A, Single-Arm Study to Evaluate the Safety and Clinical Activity of VTX2735 in Participants With Cryopyrin-Associated Periodic Syndrome (CAPS)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 7
- 试验地点
- 2
- 主要终点
- Safety and Tolerability of VTX2735
研究概览
简要总结
This is a study to understand if taking VTX2735 is safe and effective in participants diagnosed with Cryopyrin-Associated Period Syndrome (CAPS). Approximately 10 patients will take VTX2735 Dose A or VTX2735 Dose B.
The study consists of a screening/washout period of up to 28 weeks, a 2 week treatment period, a treatment withdrawal period of up to 2 weeks, another 2 week treatment period, and a 4 week follow up period. The maximum length of treatment is 4 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •At least 18 years of age
- •Diagnosis of CAPS and FCAS subtype and mild clinical phenotype
- •At least one flare during screening/washout
- •Women must not be of childbearing potential or must agree to use two methods of highly effective contraception during the study and for 30 days after the last dose of the study product
- •Men with a partner who is of childbearing potential must agree to use condoms plus another highly effective form of birth control during the study and for 90 days after the last dose of study product
排除标准
- •Unwilling to comply with washout of anti-IL-1 therapy and tolerate symptoms of disease flare
- •Moderate or severe CAPS manifestations or significant damage or any CAPS feature that presents a contraindication to washout of anti-IL-1 therapy
- •Has a history of chronic or recurrent infectious disease
- •Has a known immune deficiency or is immunocompromised
- •Has hepatitis B or hepatitis C infection, human immunodeficiency virus (HIV), acquired immunodeficiency syndrome (AIDS), or active tuberculosis (TB)
- •Has another clinically important medical disorder that would compromise safety
研究组 & 干预措施
Cohort 2
干预措施: VTX2735 (Drug)
Cohort 1
干预措施: VTX2735 (Drug)
结局指标
主要结局
Safety and Tolerability of VTX2735
时间窗: From the initial administration of VTX2735 through study completion, up to 10 weeks
Incidence and severity of treatment-emergent adverse events (TEAE), serious adverse events (SAE), and discontinuation due to adverse events
次要结局
- Number of days when any single DHAF2 symptom score is >3(From Day 1 to completion of treatment with VTX2735, up to Day 28)
- Mean change in the mean key symptom score (KSS) derived from the Daily Health Assessment Form, Second Generation (DHAF2) from baseline(From Day 1 to completion of treatment with VTX2735, up to Day 28)
- Maximum severity of any symptom score on DHAF2(From Day 1 to completion of treatment with VTX2735, up to Day 28)
- Number of days when the daily KSS is >3(From Day 1 to completion of treatment with VTX2735, up to Day 28)
- Achievement of 30%, 50%, or 75% improvement in mean KSS from baseline(From Day 1 to completion of treatment with VTX2735, up to Day 28)