NCT02515630已完成2 期
A Phase 2, Open-label, Translational Biology Study of Momelotinib in Transfusion-Dependent Subjects With Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF)
Sierra Oncology LLC - a GSK company0 个研究点目标入组 41 人开始时间: 2016年1月29日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 41
- 主要终点
- Transfusion Independence Response by Week 24
研究概览
简要总结
This study will evaluate the transfusion independence response rate in transfusion-dependent adults with myelofibrosis after treatment with momelotinib (MMB).
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
- 否
入选标准
- •Diagnosis of PMF or Post PV/ET-MF
- •Requires myelofibrosis therapy, in the opinion of the investigator
- •High risk OR intermediate-2 risk defined by dynamic international prognostic scoring system (DIPSS) OR intermediate-1 risk defined by DIPSS and associated with symptomatic splenomegaly and/or hepatomegaly
- •Transfusion dependent at baseline, defined as ≥ 4 U red blood cell (RBC) transfusion in the 8 weeks prior to first dose of MMB
- •Acceptable organ function as evidenced by the following:
- •Platelet Count ≥ 50 x 10^9/L
- •Aspartate aminotransferase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤ 3 x upper limit of normal (ULN) or AST or ALT ≤ 5 x ULN if liver is involved by disease process as judged by the investigator
- •Serum creatinine ≤ 2.0 mg/dL or calculated creatinine clearance of ≥ 60 mL/min
- •Direct bilirubin ≤ 2.0 x ULN
- •Life expectancy of > 24 weeks
- •Males and females of childbearing potential who engage in heterosexual intercourse must agree to use protocol specified method(s) of contraception
- •Lactating females must agree to discontinue nursing before MMB administration
- •Able to understand and willing to sign the informed consent form
排除标准
- •Prior splenectomy
- •Splenic irradiation within 3 months prior to the first dose of MMB
- •Prior treatment with MMB
- •Known positive status of human immunodeficiency virus (HIV)
- •Chronic active or acute viral hepatitis A, B, or C infection (testing required for hepatitis B and C), or hepatitis B or C carrier
- •Use of strong cytochrome P450 3A4 (CYP3A4) inducer within 2 weeks prior to the first dose of MMB
- •Uncontrolled intercurrent illness per protocol
- •Treatment with a Janus kinase (JAK) inhibitor within 21 days of the planned first dose of MMB
- •Presence of peripheral neuropathy ≥ Common Terminology Criteria for Adverse Events (CTCAE) Grade 2
- •Unwilling or unable to undergo a MRI per requirements in the study protocol
- •Unwilling to consent to genomics sampling
- •Note: Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Momelotinib
Experimental
MMB for 24 weeks (± 7 days)
干预措施: MMB (Drug)
结局指标
主要结局
Transfusion Independence Response by Week 24
时间窗: From baseline to Week 24
The percentage of subjects who became transfusion independent for ≥ 12 weeks at any time on study. A subject was considered transfusion independent on study if no RBC transfusion occurred in any 12-week period during the 24-week treatment period.
次要结局
- Change in Markers of Iron Metabolism and Anemia - Erythrocytes(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Hematocrit(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Ferritin(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Soluble Transferrin Receptor(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Transferrin Saturation(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Unsaturated Iron Binding Capacity(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Pharmacodynamics Biomarker - pSTAT3/tSTAT3 Ratio(On Day 1 and at Weeks 4 and 24)
- Transfusion Response Rate by Week 24(From baseline to Week 24)
- Splenic Response Rate at Week 24(Measured at Week 24)
- Response Rate in Total Symptom Score (TSS) at Week 24(Measured at Week 24)
- Change in Markers of Iron Metabolism and Anemia - Change From Baseline in Hepcidin Daily Change(At baseline, Day 1, Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Trough Hepcidin(At baseline, Day 1, Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Serum Iron(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Hemoglobin(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Total Iron Binding Capacity(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Reticulocytes(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Reticulocytes/Erythrocytes%(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Erythropoietin(At Weeks 8 and 20)
- Change in Markers of Iron Metabolism and Anemia - Platelets(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Leukocytes(At Weeks 2, 4, 8, 12, 16, 20 and 24)
- Change in Markers of Iron Metabolism and Anemia - Blasts(At Weeks 2 and 4)
- Change in Liver Iron Content(Measured at Week 24)
- Change in Pharmacodynamics Biomarker - pSTAT3(On Day 1 and at Weeks 4 and 24)
- Change in Inflammatory Markers - C-Reactive Protein (CRP)(At Weeks 2, 12 and 24)
研究者
相似试验
已完成
2 期
Study to Separately Evaluate the Activity of Talacotuzumab (JNJ-56022473) or Daratumumab in Transfusion-Dependent Participants With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) Who Are Relapsed or Refractory to Erythropoiesis-Stimulating Agent (ESA) TreatmentMyelodysplastic SyndromesNCT03011034Janssen Research & Development, LLC34
招募中
1 期
ALS20-101 Lentiviral Gene Therapy for Beta ThalassemiaBeta-ThalassemiaNCT06364774Children's Hospital of Philadelphia12
已完成
2 期
Efficacy and Safety of Convalescent Plasma in Treating COVID-19 Hospitalized PatientsCOVID-19NCT04354831Medical College of Wisconsin131
已完成
4 期
Immunomodulation Following TransfusionBlood Component TransfusionNCT00810810University of Washington287
已完成
2 期
Study of Tipifarnib in Patients With High-Risk Myelodysplastic Syndrome (MDS)Myelodysplastic SyndromeNCT00050154Johnson & Johnson Pharmaceutical Research & Development, L.L.C.82