Safety of Romiplostim (Nplate®) in Patients Who Have Failed to Achieve Platelet Engraftment Following Umbilical Cord Blood Transplant
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- Maximum tolerated dose of romiplostim
研究概览
简要总结
This is a single institution, phase I dose escalation study of weekly romiplostim post umbilical cord blood transplantation in patients who fail to achieve platelet engraftment by day +30. Engraftment is defined as a platelet count ≥ 20 x 109/L on 3 consecutive measurements without transfusion for 7 days. Romiplostim is administered at the assigned dose as 6 weekly injections beginning by day +42 post transplant. Up to 4 dose levels (4, 6, 8, and 10 mcg/kg/dose) will be evaluated with the maximum tolerated dose (MTD) of romiplostim determined by using the Continual Reassessment Method (CRM). The goal of this CRM will be to identify 1 of the 4 dose levels which corresponds to the desired maximum toxicity rate of 20% or less.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 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
- •Any patient who has undergone a single or double umbilical cord blood transplant (UCBT) except those with primary myelofibrosis.
- •Those with acute leukemia must be in remission at the time of transplant
- •Must have achieved neutrophil engraftment (defined as an ANC >500 for three consecutive days) and be off daily G-CSF prior to starting romiplostim. Intermittent G-CSF is allowed.
- •Failure to achieve platelet engraftment (defined as platelet count ≥20x10^9/L on 3 consecutive measurements without transfusion for 7 days) by day +28 post UCBT
- •Between day +28 and day +42 status post myeloablative or nonmyeloablative UCBT (single or double cord blood transplant)
- •Age ≥ 18 years
- •Adequate organ function within 7 days of enrollment defined as:
- •Creatinine: ≤ 2.0 mg/dL
- •Hepatic: SGOT and SGPT < 5 x upper limit of institutional normal (ULN)
- •Women of child bearing potential agree to use effective contraception during therapy and for 4 months after completion of therapy
- •Voluntary written consent
排除标准
- •Known pregnancy - Pregnancy Category C: there are no adequate and well-controlled studies of romiplostim in pregnancy
- •Recurrence of AML or myelodysplastic syndrome on bone marrow evaluation done within 21 days of enrollment
- •Presence of clinically significant bone marrow fibrosis on the bone marrow examination immediately prior to UCBT
- •Patients requiring more than one platelet transfusion per day
- •History of an allergy to romiplostim
研究组 & 干预措施
Romiplostim treatment
Romiplostim at the assigned dose will be administered subcutaneously (SQ) once a week for 6 weeks (i.e. 6 doses) unless platelet count exceeds 100 x 10^9/L and at least 4 doses of therapy were given.After the initial dose, subsequent doses will be administered within a window of +/- 3 days.
干预措施: Romiplostim (Drug)
结局指标
主要结局
Maximum tolerated dose of romiplostim
时间窗: Day +28 blood transplant (UCBT)
Measured by number of patients who have failed to achieve platelet engraftment by day +28 after a myeloablative or nonmyeloablative umbilical cord blood transplant (UCBT)
次要结局
- Platelet recovery(Day +28)
- Bone marrow fibrosis(Day 100 post transplant)
- Thrombocytopenia(Day +28)