Safety of Romiplostim (Nplate®) Following UCBT

NCT ID: NCT02046291

Last Updated: 2021-06-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-04-10
• Study Completion Date: 2021-06-01

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.

Conditions

Hematologic Malignancies

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Romiplostim

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Other Intervention Names

Nplate

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Margaret MacMillan, MD, MSc, FRCPC

Role: PRINCIPAL_INVESTIGATOR

University of Minnesota

Locations

University of Minnesota Masonic Cancer Center

Minneapolis, Minnesota, United States

Countries

United States

Other Identifiers

MT2012-17R

Identifier Type: OTHER

Identifier Source: secondary_id

2012LS089

Identifier Type: -

Identifier Source: org_study_id