Safety of Romiplostim (Nplate®) Following UCBT

NCT02046291 | Completed Phase 1 DMC

• 2 other identifiers: 2012LS089MT2012-17R
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Platelet recovery

  Day +28

• Thrombocytopenia

  Day +28

• Bone marrow fibrosis

  Day 100 post transplant

Study Arms (1)

Romiplostim treatment

EXPERIMENTAL

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

Interventions

Romiplostim 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.

Also known as: Nplate

Romiplostim treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota: lead

Study Sites (1)

University of Minnesota Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Hematologic Neoplasms; Hodgkin Disease; Leukemia, Lymphoid; Multiple Myeloma; Leukemia, Monocytic, Acute; Lymphoma, Non-Hodgkin

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoma; Neoplasms by Histologic Type; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Leukemia; Neoplasms, Plasma Cell; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hemorrhagic Disorders; Leukemia, Myeloid, Acute; Leukemia, Myeloid

Study Officials

• Margaret MacMillan, MD, MSc, FRCPC

  University of Minnesota

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2014
• First Posted: January 27, 2014
• Study Start: April 10, 2015
• Primary Completion: December 12, 2020
• Study Completion: June 1, 2021
• Last Updated: June 8, 2021

Record last verified: 2021-06

Locations

US (1)