AN INTERVENTIONAL PHASE 2, OPEN-LABEL, MULTI-CENTER STUDY TO EVALUATE SAFETY AND EFFICACY OF PF-06835375 IN ADULT PARTICIPANTS WITH MODERATE TO SEVERE PRIMARY IMMUNE THROMBOCYTOPENIA
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- Pfizer
- 入组人数
- 91
- 试验地点
- 59
- 主要终点
- Proportion of participants with change from baseline of platelet counts
研究概览
简要总结
This is a Phase 2, open-label, multicenter, multiple subcutaneous injection, safety and efficacy study of PF-06835375 in adult participants with primary immune thrombocytopenia (ITP). This study will focus on participants with persistent (>3 months and ≤12 months), or chronic (>12 months) ITP
详细描述
This study is designed to elucidate the effects of PF-06835375 on platelet counts in participants with moderate to severe primary ITP. Based on the experience with other B-cells depleting agents, it is expected that the platelet counts will increase following a standard treatment. Each participant in cohort 1 received 1 subcutaneous injection of dose 1 every month for 3 months during the 12-week treatment period. Each participant in cohorts 2, 3, or 4 (will) receive(d) subcutaneous injection(s) of dose 2, 3, or 4 every month for 4 months during the 16-week treatment period. This should provide sufficient levels of exposure and depletion of CXCR5 positive cells to sustain the effects of PF-06835375 during the treatment period. Additional depletion of Tfh cells may provide sustained increase in platelet count following the last treatment.
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
盲法说明
open label
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Primary ITP. Ongoing ITP (platelet counts <50 x 109/L) [No severe bleeding within 1 month or during screening] AND Persistent ITP (3 to 12 months) or Chronic ITP >12 months
排除标准
- •Bleeding event according to the WHO grading scale ≥2 occurring ≤4 weeks prior to screen OR a current bleeding event that, in the opinion of the investigator, requires treatment with standard of care therapy OR require blood or blood products during screening
- •Splenectomy within 3 months of randomization or planned during the study duration.
研究组 & 干预措施
Open Label PF-06835375 dose 1 Treatment
subcutaneous injection once monthly for 3 months
干预措施: PF-06835375 (Biological)
Open Label PF-06835375 dose 3 Treatment
subcutaneous injection once monthly for 4 months
干预措施: PF-06835375 (Biological)
Open Label PF-06835375 dose 2 Treatment
subcutaneous injection once monthly for 4 months
干预措施: PF-06835375 (Biological)
Open Label PF-06835375 dose 4 Treatment
subcutaneous injections once monthly for 4 months
干预措施: PF-06835375 (Biological)
结局指标
主要结局
Proportion of participants with change from baseline of platelet counts
时间窗: baseline through 12 and 16 weeks
To evaluate absolute value of platelet count of treated participants
次要结局
- proportion of participants with complete response (CR)(baseline through 12 and 16 weeks)
- proportion of participants with modified overall response (mOR)(baseline through 12 and 16 weeks)
- Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs)(baseline through end of study (Week 20 for cohort 1 and Week 24 for cohorts 2 and 3))
- Proportion of participants with change from baseline of platelet counts(baseline to Week 20 and Week 24)
- Proportion of participants with change from baseline of circulating B cells(baseline to Week 20 and Week 24)
- Proportion of participants with change from baseline of circulating cTfh cells(baseline to Week 20 and Week 24)