A Phase II Open-label Study of Combined Ruxolitinib and Enasidenib in Patients With Accelerated/Blast-phase Myeloproliferative Neoplasm or Chronic-phase Myelofibrosis With an IDH2 Mutation
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 11
- 主要终点
- Number of MPN Participants With Response
研究概览
简要总结
The presence of IDH mutation is associated with worse survival in patients with myelofibrosis. Moreover IDH mutations are among the most frequently encountered events in MPNs that have progressed to acute myeloid leukemia. Ruxolitinib, a JAK1/2 inhibitor, and enasidenib an IDH2 inhibitor are effective and tolerable treatments for patients with myelofibrosis (MF) and acute myeloid leukemia (AML), respectively. The study team hypothesize that the combination of these agents in patients with MPN with an IDH2 mutation will improve the overall clinical response to therapy.
详细描述
At this time, there is no standard medical treatment for MPN-AP/BP and most patients with accelerated and blast phase MPN do not respond well to treatment This is a phase II open-label study to evaluate the safety and efficacy of combined ruxolitinib and enasidenib in patients with accelerated/blast-phase myeloproliferative neoplasm or chronic phase myelofibrosis with high risk features and IDH2 mutation.
Ruxolitinib (Jakafi/Jakavi) is FDA approved for myelofibrosis and was shown to reduce splenomegaly and improve symptoms. Enasidenib is a potent inhibitor of the IDH2 mutant enzyme and is FDA approved for relapsed refractory AML where it showed effectivity.
Pre-clinical studies indicate increased disease mitigating effects with the combination of enasidenib and ruxolitinib.
This study will enroll up to 32 patients. Ruxolitinib and enasidenib will be given orally in 28-day cycles.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Patients with MPN
Ruxolitinib and Enasidenib combination therapy
干预措施: Ruxolitinib (Drug)
Patients with MPN
Ruxolitinib and Enasidenib combination therapy
干预措施: Enasidenib (Drug)
结局指标
主要结局
Number of MPN Participants With Response
时间窗: 6 Months
The number of treated accelerated-phase and blast-phase MPN patients (primary cohort) that achieve a best response per 2013 International Working Group (IWG) criteria of either complete response (CR), Partial Response (PR), or complete response with incomplete recovery of counts (CRi), when treated with the combination of ruxolitinib with enasidenib within 6 cycles of combined therapy. Complete Response with incomplete recovery of counts (CRi) - complete remission (\<5% marrow blasts by morphology) with incomplete count recovery (platelet count \<100 x 10\^9\^/L and/or absolute neutrophil count \< 1 x 10\^9\^/L) Complete Response (CR) - full marrow recovery; full count recovery; resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH Partial Response (PR) - morphologic remission in the peripheral blood but not necessarily in the bone marrow; resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH
次要结局
- Number of MPN Participants With Blast Response(6 Months)
- Number of MF-CP Participants With Any Response(6 Months)
研究者
John Mascarenhas
Associate Professor
Icahn School of Medicine at Mount Sinai