Eltrombopag for Thrombocytopenia in Patients With Relapsed Multiple Myeloma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01484314
Recruitment Status : Terminated (Low accrual.)
First Posted : December 2, 2011
Results First Posted : November 17, 2016
Last Update Posted : March 15, 2017
Information provided by (Responsible Party):
Nancy Berliner, MD, Dana-Farber Cancer Institute

Brief Summary:

Eltrombopag is a compound that may help stimulate the production of platelets. This drug has been used in treatment of low platelet counts caused by a disorder called idiopathic thrombocytopenic purpura and information from those other research studies suggests that Eltrombopag may help to maintain platelet counts in patients with relapsed multiple myeloma in this research study.

In this research study,the investigators are trying to determine if Eltrombopag is effective in maintaining platelet counts in patients who are being treated for relapsed multiple myeloma.

Condition or disease Intervention/treatment Phase
Thrombocytopenia Multiple Myeloma Drug: Eltrombopag Phase 2

Detailed Description:

You will take eltrombopag during two consecutive cycles of chemotherapy. During these two cycles you will be asked to complete the following for the research study in addition to any clinical exams or procedures that your regular doctor may order:

Study Drug(s): If you take part in this research study, you will be given a study drug-dosing calendar for each treatment cycle. Each treatment cycle lasts 27 days during which time you will be taking the study drug for 11 days on days 1 through 11.

Clinical Exams: You will have a physical exam on days 1, 11, and 21 of both cycles. You will be asked questions about your general health and specific questions about any problems that you might be having and any medications you may be taking. Your vital signs will be collected during this exam.

Performance status: Your ability to perform daily tasks will be assessed on days 1 and 27 of both cycles.

Blood Tests: You will have tests to check for safety performed on days 1, 7, 11, 20, and 27 of both cycles. You will have approximately 1-2 teaspoons of blood collected.

Concurrent Medications: Any changes to your medications will be recorded. Adverse Events: You be continually monitored for any changes to you health or well-being while you are on this study.

You will complete all subsequent cycles of chemotherapy procedures per standard of care for DFCI. During the subsequent cycles, you will not take eltrombopag.

After the final dose of the study drug: On Day 1 of cycle 3 of chemotherapy, you will complete the end of study/off study evaluations. The following procedures will be completed:

  • Physical Exam, including vitals signs
  • Performance status, which evaluates how you are able to carry on with your usual activities.
  • Tumor Measurements: We will assess your tumor by blood tests and/or bone marrow aspirate and biopsy. If clinically indicated, you may have CT scans, bone scans or skeletal surveys
  • Blood tests, routine tests to check for safety
  • Concurrent Medications: Any changes to your medications will be recorded.


We would also like to keep track of your medical condition for thirty days after your last dose of study drug. We would like to do this by having you return to have your blood counts checked once a week for 4 weeks and by calling you to check your condition and any changes to your health or well-being that may have occurred since you last took the study drug.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study of Eltrombopag for the Treatment of Thrombocytopenia in Patients Undergoing Therapy for Relapsed Multiple Myeloma
Actual Study Start Date : April 2012
Actual Primary Completion Date : September 2014
Actual Study Completion Date : September 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Multiple Myeloma
Drug Information available for: Eltrombopag
U.S. FDA Resources

Arm Intervention/treatment
Experimental: Migration Arm
Administration of eltrombopag to support platelets during chemotherapy
Drug: Eltrombopag
100mg (patients of East Asian descent will receive a dose of 50mg) orally dosed once daily starting 6 days prior to initiation of chemotherapy for a total of 11 days of treatment during two consecutive cycles of chemotherapy

Primary Outcome Measures :
  1. Maintenance of Platelet Count [ Time Frame: 2 years ]
    To determine the proportion of patients with relapsed multiple myeloma in whom eltrombopag maintains platelet counts at > 90% of baseline platelet counts with no more than 4 units of platelet transfusions at day 1 of Cycle 3 of chemotherapy

  2. Safety and Tolerability [ Time Frame: 2 years ]
    To determine whether eltrombopag administration results in an increased number of participants with adverse events.

Secondary Outcome Measures :
  1. Incidence of Grade 3/4 Thrombocytopenic Events [ Time Frame: 2 years ]
    To assess whether eltrombopag decreases the incidence of grade 3/4 thrombocytopenic events by day 1 of Cycle 3 of chemotherapy

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Participants must have histologically confirmed multiple myeloma that is relapsed or refractory
  • Patients may have had 1 or more prior chemotherapy regimens for multiple myeloma but none within the preceding 14 days
  • Performance status (ECOG) ≤ 2
  • Not pregnant or breastfeeding

Exclusion Criteria:

  • Participants with a history of rapidly progressive disease [increase in tumor size (≥ 50%) as defined by standard myeloma markers ], pelvic irradiation, chemotherapy or radiotherapy within the preceding 14 days
  • Participants may not be receiving any other study agents within 21 days prior to entry on the study.
  • The use of growth factors other than erythropoiesis stimulating agents or G-CSF (Neupogen or Neulasta) during the study period.
  • Participants with evidence of active thromboembolic disease or a history of thromboembolism within the preceding 6 months (excluding thrombosis of a central line).
  • Participants with a documented history of genetic predisposition for thrombosis (anti-phospholipid antibody syndrome, AT-III deficiency, etc.), platelet disorder or bleeding disorder.
  • Uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Individuals with a history of other malignancies are eligible only if they have been disease-free for at least 5 years and are deemed by the investigator to be at low risk for recurrence of that malignancy. Individuals with the following cancers are eligible if diagnosed and treated within the preceding 5 years: cervical cancer in situ, and basal cell or squamous cell carcinoma of the skin.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01484314

United States, Massachusetts
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Sponsors and Collaborators
Dana-Farber Cancer Institute
Principal Investigator: Nancy Berliner, MD Dana-Farber Cancer Institute

Responsible Party: Nancy Berliner, MD, Principal Investigator, Dana-Farber Cancer Institute Identifier: NCT01484314     History of Changes
Other Study ID Numbers: 11-373
First Posted: December 2, 2011    Key Record Dates
Results First Posted: November 17, 2016
Last Update Posted: March 15, 2017
Last Verified: February 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Nancy Berliner, MD, Dana-Farber Cancer Institute:

Additional relevant MeSH terms:
Multiple Myeloma
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Blood Platelet Disorders