Safety of Romiplostim (Nplate®) Following UCBT
- Registration Number
- NCT02046291
- Lead Sponsor
- Masonic Cancer Center, University of Minnesota
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 21
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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
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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.
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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
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Between day +28 and day +42 status post myeloablative or nonmyeloablative UCBT (single or double cord blood transplant)
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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)
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Women of child bearing potential agree to use effective contraception during therapy and for 4 months after completion of therapy
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Voluntary written consent
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Romiplostim treatment Romiplostim 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.
- Primary Outcome Measures
Name Time Method Maximum tolerated dose of romiplostim 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)
- Secondary Outcome Measures
Name Time Method Platelet recovery Day +28 Average speed of platelet recovery in patients who have failed to achieve platelet engraftment.
Bone marrow fibrosis Day 100 post transplant Incidence of bone marrow fibrosis.
Thrombocytopenia Day +28 Incidence of clinically significant bleeding episodes and number of platelet transfusions.
Trial Locations
- Locations (1)
University of Minnesota Masonic Cancer Center
🇺🇸Minneapolis, Minnesota, United States