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Safety of Romiplostim (Nplate®) Following UCBT

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02046291
Recruitment Status : Active, not recruiting
First Posted : January 27, 2014
Last Update Posted : April 21, 2020
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Hematologic Malignancies Drug: Romiplostim Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official Title: Safety of Romiplostim (Nplate®) in Patients Who Have Failed to Achieve Platelet Engraftment Following Umbilical Cord Blood Transplant
Actual Study Start Date : April 10, 2015
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : June 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Romiplostim

Arm Intervention/treatment
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.
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.
Other Name: Nplate

Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. Platelet recovery [ Time Frame: Day +28 ]
    Average speed of platelet recovery in patients who have failed to achieve platelet engraftment.

  2. Thrombocytopenia [ Time Frame: Day +28 ]
    Incidence of clinically significant bleeding episodes and number of platelet transfusions.

  3. Bone marrow fibrosis [ Time Frame: Day 100 post transplant ]
    Incidence of bone marrow fibrosis.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 ≥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

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02046291

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United States, Minnesota
University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
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Principal Investigator: Angela Smith, MD University of Minnesota
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Responsible Party: Masonic Cancer Center, University of Minnesota Identifier: NCT02046291    
Other Study ID Numbers: 2012LS089
MT2012-17R ( Other Identifier: University of Minnesota Blood and Marrow Transplant Program )
First Posted: January 27, 2014    Key Record Dates
Last Update Posted: April 21, 2020
Last Verified: April 2020
Keywords provided by Masonic Cancer Center, University of Minnesota:
umbilical cord transplant
acute leukemia
Hodgkin's Lymphoma
Lymphoid Leukemia
Multiple Myeloma
Myeloid and Monocytic Leukemia
Non-Hodgkin's Lymphoma
Hematopoietic malignancies
Additional relevant MeSH terms:
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Hematologic Neoplasms
Neoplasms by Site
Hematologic Diseases