To study the safety and efficacy of oral Desidustat for treatment of anemia in patients with Lower risk Myelodysplastic syndrome [MDS] and Myeloproliferative neoplasms [MPN]
试验速览
- 阶段
- Phase 3 4
- 状态
- 尚未招募
- 发起方
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- The primary efficacy endpoint will be the proportion of patients who were TI for more than or equal to 8 consecutive weeks during the first 28 treatment weeks.
研究概览
简要总结
Background: Anemia is the predominant symptom in lower risk myelodysplastic syndrome (LR-MDS) and some myeloproliferative illnesses especially primary myelofibrosis [MPN]. Response to standard medicines is seen in only 30-40% of patients and many patients end up requiring multiple transfusions. Desidustat is a novel hypoxia inducing factor [HIF-PHD] inhibitor which is used mainly for the treatment of anemia in patients with chronic kidney disease [both dialysis dependent and non-dependent]. It mimics the body’s natural response to the hypoxic condition by inhibiting HIF-PH, thus preventing hydroxylation of HIF-alpha and allowing for the transcription and expression of genes necessary for erythropoiesis, such as EPO and iron metabolism factors. Based on the effectiveness of Desidustat in the treatment of anemia in CKD, we hypothesized a potential clinical benefit in anemia of LR-MDS and MPN.
Methodology: This is asingle arm, Phase II, Safety and Efficacy trial where we will study the efficacy of Desidustat in treating transfusion dependent anemia in patients with lower risk MDS or MPN. Desidustat will be orally administered 100 mg 3 days in a week for 6 months. If there is inadequate response after 2 months of initiation of therapy, the dose will be increased to 150mg three days a week for the subsequent 4 months, along with best supportive care. We will also collect blood samples from patients at 0, 2 and 6 months to look for HIFa expression, erythropoietin levels and iron parameters.
Results: The primary efficacy endpoint will be the proportion of patients with transfusion independence (TI) for more than 8 consecutive weeks in the first 24 treatment weeks. Secondary efficacy endpoint will be the proportion of patients with a more than 50% reduction in RBC transfusions over an 8-week period compared with baseline. We will also study if HIFa expression correlated with hematological response.
Conclusions: We hope to show that Desidustat will be able to ameliorate anemia in a group of patients with MDS and MPN.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 盲法
- None
入排标准
- 年龄范围
- 18.00 Year(s) 至 75.00 Year(s)（—）
- 性别
- All
入选标准
- •a)Very low, low, or intermediate-risk MDS based on IPSS-R score with less than 5% bone marrow blasts; baseline Hb of less than or equal to 9 g/dL; b)MPN with anemia; c)18 years and above and d)Low RBC transfusion burden, defined as 1–4 packed red blood cell (pRBC) units per 8-week period or 1 pRBC transfusion per 8-week period for 2 consecutive 8-week periods before randomization.
排除标准
- •a)Higher – risk MDS; Del 5q cytogenetic abnormality; or have anemia of a non-MDS etiology (e.g., iron deficiency).
- •b)Children c)Patients who are unable to come for a visit atleast every 2 months in the initial 6 month period of the study.
结局指标
主要结局
The primary efficacy endpoint will be the proportion of patients who were TI for more than or equal to 8 consecutive weeks during the first 28 treatment weeks.
时间窗: 2 months | 6 months | 12 months | 18 months | 24 months
次要结局
- (1) Study the proportion of patients who had above 50% reduction in the number of RBC transfusions over any 8 weeks compared with baseline,((2) Study the proportion of patients who were TI for more than 20 consecutive weeks.)
研究者
Archita R
Christian Medical College Vellore Ranipet campus