Using Romiplostim to Treat Low Platelet Counts Following Chemotherapy and Autologous Hematopoietic Cell Transplantation in People With Blood Cancer

An Open-Label, Pilot Study of Romiplostim for Conditioning Regimen-Related Thrombocytopenia After High-Dose Therapy and Autologous Hematopoietic Cell Transplantation

The purpose of this study is to see if the study drug, romiplostim, helps low platelet count caused by the standard blood cancer treatment of chemotherapy and autologous hematopoietic cell transplantation. This study will also look at whether romiplostim can decrease the number of times the participant needs to return to the clinic for platelet transfusions to treat their low platelet count. In addition, the researchers will determine how safe it is to give romiplostim to people with blood cancer who have received treatment with chemotherapy and autologous hematopoietic cell transplantation.

Study Overview

• Status: Completed
• Conditions: Multiple Myeloma; Hodgkin Lymphoma; Non-Hodgkin Lymphoma; HDT-AHCT
• Intervention / Treatment: Drug: Romiplostim

• Study Type: Interventional
• Enrollment (Actual): 62
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • Basking Ridge, New Jersey, United States, 07920
      • Memorial Sloan Kettering at Basking Ridge (Limited Protocol Activities)
    • Middletown, New Jersey, United States, 07748
      • Memorial Sloan Kettering Monmouth (Limited Protocol Activities)
    • Montvale, New Jersey, United States, 07645
      • Memorial Sloan Kettering Bergen (Limited Protocol Activities)
  • New York
    • Commack, New York, United States, 11725
      • Memorial Sloan Kettering Commack (Limited protocol activities)
    • Harrison, New York, United States, 10604
      • Memorial Sloan Kettering Westchester (Limited Protocol Activities)
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center (All Protocol Activities)
    • Uniondale, New York, United States, 11553
      • Memorial Sloan Kettering Nassau (Limited Protocol Activities)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Adult patients ≥ 18 years old diagnosed with multiple myeloma (MM), any subtype of Hodgkin lymphoma (HL), or any subtype of non-Hodgkin lymphoma (NHL)

  • For MM, the conditioning regimen used will be high-dose melphalan.°For HL and NHL, the conditioning will be one of the following high-dose regimens: BEAM, CBV, or TBC.
  • Other conditioning regimens not listed above, or variations of the above conditioning regimens, may be allowed at the discretion of the principal investigator if the regimen is considered myeloablative.

• Adequate organ function is required, defined as follows:

  • Serum bilirubin ≤ 2 mg/dL, unless benign congenital hyperbilirubinemia.
  • AST, ALT, and alkaline phosphatase < 3 times the upper limit of normal.
  • Creatinine clearance ≥ 40 ml/min (calculated by Cockcroft Gault)
  • LVEF ≥ 45% by MUGA or resting echocardiogram
  • Pulmonary function (FEV1 and corrected DLCO) ≥ 45% predicted.
  • Adequate performance status ECOG ≤ 2.
• Ability to provide written informed consent.
• Patients undergoing HDT-AHCT.

Exclusion Criteria:

• Patients with a previous diagnosis of a myeloid malignancy.
• Patients for whom the treating oncologist will be using a non-standard platelet transfusion threshold during the AHCT.

• Patients with a history of a prior symptomatic or incidental venous thromboembolic event (such as DVT or pulmonary embolism) within the prior 6 months are eligible if they are on and tolerating anti-coagulation, or greater than 6 months ago are eligible if they completed or are on and tolerating anti-coagulation.

  °A venous thrombotic event associated with a central venous catheter will not make the patient ineligible.

• Patients with a history of symptomatic arterial thrombotic events such as myocardial infarction, ischemic cerebral vascular accident or transient ischemic attack in the past 6 months are ineligible.
• Patients who had been diagnosed with Immune Thrombocytopenic Purpura (ITP) at any time prior to the AHCT are ineligible.
• Patients with a serious concomitant medical condition that could interfere with the conduct of the clinical trial, such as unstable angina, renal failure requiring hemodialysis, or active infection requiring IV antibiotics.
• Previous use of romiplostim, PEGylated recombinant human megakaryocyte growth and development factor, eltrombopag, recombinant human TPO, any other TPO receptor agonist, or any investigational platelet producing agent.
• Females who are pregnant or breastfeeding or planning to become pregnant or breastfeed during treatment and for an additional 30 days after treatment discontinuation or longer if required by prescribing information for chemotherapy received during the study.
• Patients unwilling to use highly effective contraception during the study period and for the duration required by prescribing information for chemotherapy(ies) administered during the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: romiplostim; Patients will be enrolled prior to admission for High-Dose Therapy and Autologous Hematopoietic Cell Transplantation (HDT-AHCT), and they will undergo their planned HDT-AHCT for their respective hematologic malignancy as per institutional standards. Regardless of the conditioning regimen received, all patients will receive romiplostim 3.0 mcg/kg SC on Day +1 and romiplostim 2.0 mcg/kg SC on Day +8 after HDT-AHCT. Beyond Day +8, patients will be treated until platelet count is >50,000/mcL, without any platelet transfusions in the prior 48 hours. All doses after the second romiplostim dose will be titrated as per Table 3, based on weekly CBC/platelet counts. No patient will receive more than six doses of romiplostim, even if platelets have not corrected by Day +42.

Drug: Romiplostim; Romiplostim 3.0 mcg/kg SC on Day +1 and Romiplostim 2.0 mcg/kg SC on Day +8 after HDT-AHCT. Beyond Day +8, patients will be treated weekly until platelet count is >50,000/mcL, without any platelet transfusions in the prior 48 hours. All doses after the second Romiplostim dose will be titrated as per Table 3, based on weekly CBC/platelet counts. Patients will receive a maximum of six weekly doses of romiplostim.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Days Post HDT-AHCT Requiring Transfusions or Grade 4 CTCAE Thrombocytopenia	Common Terminology Criteria for Adverse Events (CTCAE) Version 5.	up to 42 days

Secondary Outcome Measures

Outcome Measure	Time Frame
Number of Platelet Transfusions Per Participant Issued During the AHCT Admission	up to 100 days from AHCT

Collaborators and Investigators

• Sponsor: Memorial Sloan Kettering Cancer Center
• Collaborators: Amgen
• Investigators: Principal Investigator: Michael Scordo, MD, Memorial Sloan Kettering Cancer Center

Publications and helpful links

Helpful Links

• Memorial Sloan Kettering Cancer Center

Study record dates

Study Major Dates

• Study Start (Actual): July 14, 2020
• Primary Completion (Actual): June 8, 2023
• Study Completion (Actual): June 8, 2023

Study Registration Dates

• First Submitted: July 15, 2020
• First Submitted That Met QC Criteria: July 17, 2020
• First Posted (Actual): July 20, 2020

Study Record Updates

• Last Update Posted (Actual): February 14, 2024
• Last Update Submitted That Met QC Criteria: January 23, 2024
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Keywords: Thrombocytopenia; Autologous Hematopoietic Cell Transplantation; Romiplostim; High-Dose Therapy; 20-180
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Hematologic Diseases; Hemorrhagic Disorders; Hemostatic Disorders; Paraproteinemias; Blood Protein Disorders; Neoplasms, Plasma Cell; Lymphoma; Multiple Myeloma
• Other Study ID Numbers: 20-180

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No