A Phase 2/3 Trial of FG-4592 for Treatment of Anemia in Subjects With Lower Risk Myelodysplastic Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- FibroGen
- 入组人数
- 43
- 试验地点
- 38
- 主要终点
- Percentage of Participants With a Hemoglobin (Hb) Response to FG-4592 Without Transfusion
研究概览
简要总结
The objective of this study to evaluate the efficacy FG-4592 for the treatment of anemia in Chinese participants with lower risk MDS.
详细描述
This is a Phase 2/3 trial with sequential Phase 2 and Phase 3 components. In each part, there is an up to 4 weeks screening period followed by a treatment period of 26 weeks and a 4 week follow-up period.
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
Phase 2 part is an open label, single arm, dose-escalation study. Phase 3 component is a randomized, double blind, placebo-controlled study.
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
- •Diagnosis of primary MDS classified as very low, low or intermediate risk with <5% blasts (documented within 12 weeks prior to Day 1)
- •Screening Hb <10 g/dL and ≥6g/dL
- •Transfusion independent defined as no red blood cell transfusions within 12 weeks of Day 1
- •Erythropoiesis-stimulating agent (ESA)-naïve (not within 30 days of Day 1)
- •Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2
排除标准
- •Diagnosis of secondary MDS
- •Significant myelofibrosis (>2+fibrosis)
- •Prior therapy with azacitidine, decitabine, antithymocyte globulin, cyclosporine, thalidomide, or lenalidomide within 12 weeks prior to Day 1
- •Baseline erythropoietin level of >400 units (U)/liter (L)
- •Clinically significant anemia due to non-MDS etiologies
- •Note: Other inclusion and exclusion criteria may apply.
研究组 & 干预措施
Phase 2 Part: FG-4592
Fixed starting doses (different doses for lower body weight & higher body weight) of FG-4592 administered orally 3 times a week (TIW) for up to 26 weeks; dose adjustments to Hb levels are allowed during the study.
干预措施: FG-4592 (Drug)
Phase 3 Part: FG-4592
Fixed starting doses (different doses for lower body weight & higher body weight) of FG-4592 administered orally TIW for up to 26 weeks; dose adjustments to Hb levels are allowed during the study.
干预措施: FG-4592 (Drug)
Phase 3 Part: Placebo
Placebo (matching to FG-4592) administered orally TIW for up to 26 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Percentage of Participants With a Hemoglobin (Hb) Response to FG-4592 Without Transfusion
时间窗: 26 weeks
Hemoglobin response to FG-4592 is defined as an increase in mean Hb of ≥1.0 grams (g)/deciliter (dL) from baseline within any 8-week period during the study without transfusion.
次要结局
- Number of Participants With Hb Increase ≥1.0 g/dL From Baseline(26 weeks)
- Time to First Hb Response(26 weeks)
- Change From Baseline in Mean Hb in Weeks 25 to 27(Baseline, Weeks 25 to 27)
- Percentage of Participants With Mean Hb ≥10.0 g/dL Within Any 8-week Period During the Study Without Transfusion(26 weeks)
- Number of Participants that Require Transfusions or Hb <6 g/dL up to Week 27(Up to Week 27)
- Mean Change From Baseline in Functional Assessment of Cancer Therapy - Anemia (FACT-An) and Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-F) Scores at Week 27(30 weeks)