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Safety of Romiplostim (Nplate®) Following UCBT

Primary Purpose

Hematologic Malignancies

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Romiplostim

About this trial

This is an interventional supportive care trial for Hematologic Malignancies focused on measuring umbilical cord transplant, acute leukemia, UCBT, Hodgkin's Lymphoma, Lymphoid Leukemia, Multiple Myeloma, Myeloid and Monocytic Leukemia, Non-Hodgkin's Lymphoma, Hematopoietic malignancies

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

University of Minnesota Masonic Cancer Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Romiplostim treatment

Arm Description

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.

Outcomes

Primary Outcome Measures

Maximum tolerated dose of romiplostim

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

Platelet recovery

Average speed of platelet recovery in patients who have failed to achieve platelet engraftment.

Thrombocytopenia

Incidence of clinically significant bleeding episodes and number of platelet transfusions.

Bone marrow fibrosis

Incidence of bone marrow fibrosis.

Full Information

NCT ID

NCT02046291

First Posted

January 23, 2014

Last Updated

June 5, 2021

Sponsor

Masonic Cancer Center, University of Minnesota

1. Study Identification

Unique Protocol Identification Number

NCT02046291

Brief Title

Safety of Romiplostim (Nplate®) Following UCBT

Official Title

Safety of Romiplostim (Nplate®) in Patients Who Have Failed to Achieve Platelet Engraftment Following Umbilical Cord Blood Transplant

Study Type

Interventional

2. Study Status

Record Verification Date

June 2021

Overall Recruitment Status

Completed

Study Start Date

April 10, 2015 (Actual)

Primary Completion Date

December 12, 2020 (Actual)

Study Completion Date

June 1, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Masonic Cancer Center, University of Minnesota

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hematologic Malignancies

Keywords

umbilical cord transplant, acute leukemia, UCBT, Hodgkin's Lymphoma, Lymphoid Leukemia, Multiple Myeloma, Myeloid and Monocytic Leukemia, Non-Hodgkin's Lymphoma, Hematopoietic malignancies

7. Study Design

Primary Purpose

Supportive Care

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

21 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Romiplostim treatment

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Romiplostim

Other Intervention Name(s)

Nplate

Intervention Description

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.

Primary Outcome Measure Information:

Title

Maximum tolerated dose of romiplostim

Description

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)

Time Frame

Day +28 blood transplant (UCBT)

Secondary Outcome Measure Information:

Title

Platelet recovery

Description

Average speed of platelet recovery in patients who have failed to achieve platelet engraftment.

Time Frame

Day +28

Title

Thrombocytopenia

Description

Incidence of clinically significant bleeding episodes and number of platelet transfusions.

Time Frame

Day +28

Title

Bone marrow fibrosis

Description

Incidence of bone marrow fibrosis.

Time Frame

Day 100 post transplant

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Margaret MacMillan, MD, MSc, FRCPC

Organizational Affiliation

University of Minnesota

Official's Role

Principal Investigator

Facility Information:

Facility Name

University of Minnesota Masonic Cancer Center

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55455

Country

United States

12. IPD Sharing Statement

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Safety of Romiplostim (Nplate®) Following UCBT

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