Safety of Romiplostim (Nplate®) Following UCBT

Overview

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.

Full Title of Study: “Safety of Romiplostim (Nplate®) in Patients Who Have Failed to Achieve Platelet Engraftment Following Umbilical Cord Blood Transplant”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Supportive Care
    • Masking: None (Open Label)
  • Study Primary Completion Date: December 12, 2020

Interventions

  • Drug: Romiplostim
    • Romiplostim at the assigned dose will be administered subcutaneously (SQ) once a week for 6 weeks. The dose will be assigned at the time of subject registration. This is a dose escalation to determine the maximum tolerated dose. The doses include 4 mcg/kg/dose, 6 mcg/kg/dose, 8 mcg/kg/dose, and 10 mcg/kg/dose.

Arms, Groups and Cohorts

  • Experimental: 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.

Clinical Trial Outcome Measures

Primary Measures

  • Maximum tolerated dose of romiplostim
    • Time Frame: 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 Measures

  • Platelet recovery
    • Time Frame: Day +28
    • Average speed of platelet recovery in patients who have failed to achieve platelet engraftment.
  • Thrombocytopenia
    • Time Frame: Day +28
    • Incidence of clinically significant bleeding episodes and number of platelet transfusions.
  • Bone marrow fibrosis
    • Time Frame: Day 100 post transplant
    • Incidence of bone marrow fibrosis.

Participating in This Clinical Trial

Inclusion Criteria

  • 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 ≥20×10^9/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 Exclusion Criteria:

  • 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

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Masonic Cancer Center, University of Minnesota
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Margaret MacMillan, MD, MSc, FRCPC, Principal Investigator, University of Minnesota

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