2024-520133-72-00招募中2 期
Eltrombopag for the treatment of thrombocytopenia due to low - and intermediate risk myelodysplastic syndromes.
Associazione Qol-One9 个研究点 分布在 1 个国家目标入组 160 人开始时间: 2025年2月5日最近更新:
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 160
- 试验地点
- 9
- 主要终点
- Phase 1. Proportion of patients obtaining CR or R during the six month treatment period.
研究概览
简要总结
Phase 1 Primary Objectives To evaluate the effect of treatment relative to placebo on:
- response rate: the proportion of patients achieving a complete response (CR) or response (R) during the treatment period
- safety and tolerability in terms of frequency of adverse events (AE) and serious adverse events (SAE).
Phase 2 Primary objectives To evaluate the effect of treatment relative to placebo on:
- duration of platelet response;
- long-term safety and tolerability
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Adult subjects (18 years of age or older) with low or intermediate-1 IPSS risk MDS and stable disease.
- •Subject is practicing an acceptable method of contraception. Female subjects (or female partners of male subjects) must either be of non-childbearing potential (hysterectomy, bilateral oophorectomy, bilateral tubal ligation or post-menopausal >1 year), or of childbearing potential and use of an highly effective method of contraception from 2 weeks prior to administration of study medication, throughout the study, and 28 days after completion or premature discontinuation from the study.
- •Subjects must have a platelet count taken within the 4 weeks prior to randomization that is <30 Gi/L.
- •Subjects must be ineligible or relapsed or refractory to receive other treatment options (such as azacitidine or lenalidomide) and must be ineligible to receive intensive chemotherapy or autologous/allogeneic stem cell transplantation.
- •Subjects must have platelet count and platelet transfusion data available over a period of 8 weeks prior to randomization.
- •During the 2 months prior to randomization, subjects must have a baseline BM examination which includes cytomorphology and cytogenetics. Histopathology should be performed.
- •Erythropoiesis-stimulating agents (ESAs) in anemic subjects or granulocyte colony stimulating factor (G-CSF) in subjects with severe neutropenia and recurrent infections are allowed during the study as per accepted standards. Subjects who enter the study on ESAs or G-CSF should continue at the same dose schedule until the optimal dose of study medication has been established.
- •ECOG Performance Status 0-
- •Subject is able to understand and comply with protocol requirements and instructions.
- •Adequate baseline organ function defined by the criteria below: total bilirubin (except for Gilbert’s Syndrome) ≤ 1.5xULN ALT and AST ≤ 3xULN creatinine ≤ 2xULN albumin must not be below the lower limit of normal by more than 20%.
排除标准
- •MDS with intermediate-2 or high IPSS risk.
- •Treatment with an Investigational Product within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication.
- •Active and uncontrolled infections.
- •Subjects infected with Hepatitis B, C or Human Immunodeficiency Virus (HIV).
- •History of treatment for cancer other than MDS with systemic chemotherapy and/or radiotherapy within the last 2 years.
- •History of treatment with romiplostim or other TPO-R agonists.
- •Pre-existing cardiovascular disease (including congestive heart failure, New York Heart Association [NYHA] Grade III/IV), or arrhythmia known to increase the risk of thromboembolic events (e.g. persistent atrial fibrillation), or subjects with a QTc >450 msec (QTc >480 msec for subjects with Bundle Branch Block)
- •BM fibrosis that leads to an inability to aspirate marrow for assessment
- •Peripheral monocytosis > 1000/uL prior to Day 1 of study medication.
- •Leukocytosis >=25,000/uL prior to Day 1 of study medication.
- •Female subjects who are nursing or pregnant (positive serum or urine Beta-human chorionic gonadotropin [B-hCG] pregnancy test) at screening or pre-dose on Day
- •Current alcohol or drug abuse.
结局指标
主要结局
Phase 1. Proportion of patients obtaining CR or R during the six month treatment period.
Phase 1. Proportion of patients obtaining CR or R during the six month treatment period.
Phase 1. Safety and tolerability parameters including non-hematological laboratory Grade 3/Grade 4 toxicities, change in bone marrow blast counts from baseline and adverse events.
Phase 1. Safety and tolerability parameters including non-hematological laboratory Grade 3/Grade 4 toxicities, change in bone marrow blast counts from baseline and adverse events.
Phase 2. Duration of platelet response.
Phase 2. Duration of platelet response.
Phase 1. Long-term safety and tolerability.
Phase 1. Long-term safety and tolerability.
次要结局
- Duration of response during the treatment and follow-up periods.
- Changes in quality of life (QoL) scores.
- Frequency of platelet transfusions during the treatment and follow-up periods.
- Duration of platelet transfusion independence.
- Difference in time to response (time from starting treatment to time of achievement of CR or R).
- Incidence and severity of bleeding using the WHO Bleeding Scale.
- OS and LFS.
- Eltrombopag population pharmacokinetic parameters and plasma concentration data. The relationship between eltrombopag pharmacokinetics and relevant safety and efficacy endpoints will be explored, as data permit.
- Phase 1 primary endpoints have been reached and have been publish (Lancet Haematol. 2017 Mar;4(3):e127-e136)
研究者
Esther Natalie Oliva
Scientific
Associazione Qol-One
研究点 (9)
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