Safety of Romiplostim (Nplate®) Following UCBT

Hematologic Malignancies Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02046291
• Other study ID #: 2012LS089
• Secondary ID: MT2012-17R
• Status: Completed
• Phase: Phase 1
• Start date: April 10, 2015
• Est. completion date: June 1, 2021

Study information

• Verified date: June 2021
• Source: Masonic Cancer Center, University of Minnesota
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: June 1, 2021
• Est. primary completion date: December 12, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hematologic Malignancies
• Hematologic Neoplasms
• Neoplasms

Intervention

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.

Locations

Country	Name	City	State
United States	University of Minnesota Masonic Cancer Center	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Masonic Cancer Center, University of Minnesota

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)	Day +28 blood transplant (UCBT)
Secondary	Platelet recovery	Average speed of platelet recovery in patients who have failed to achieve platelet engraftment.	Day +28
Secondary	Thrombocytopenia	Incidence of clinically significant bleeding episodes and number of platelet transfusions.	Day +28
Secondary	Bone marrow fibrosis	Incidence of bone marrow fibrosis.	Day 100 post transplant