Clinical Trial: Phase I Study of Eltrombopag for Promoting Thrombopoiesis After Total Body Irradiation

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Phase I Study of Eltrombopag for Promoting Thrombopoiesis in Patients Undergoing Stem Cell Transplantation After Total Body Irradiation

Brief Summary:

Patients who undergo total body irradiation (TBI) for stem cell transplantation have prolonged periods of low counts of specific blood cells called platelets. These low platelets counts can cause bleeding and infection. Thus far, no drug is available for use to speed the recovery of platelets, and therefore transfusions are often necessary.

The purpose of this study is to test the safety of a drug called eltrombopag in patients who have received TBI. The investigators want to find out what effects, good or bad, it has on people with low platelet counts due to treatment with TBI. The investigators will also be testing how well eltrombopag may work at different doses and determine if this drug speeds up the recovery of the platelets.


Detailed Summary:
Sponsor: Jane Liesveld

Current Primary Outcome: Maximum Tolerated Dose (MTD) of Eltrombopag [ Time Frame: 1.5 years ]

The MTD was defined as the highest dose if no dose limiting toxicity was observed, or the highest dose at which less than one-third of the patients experienced toxicities not expected in the standard stem cell transplantation setting.


Original Primary Outcome: to determine the safety, side effect profile, and toxicity of eltrombopag when administered to patients undergoing stem cell transplantation from autologous or allogeneic donor sources, who have received myeloablative doses of TBI [ Time Frame: 1.5 years ]

Current Secondary Outcome:

  • Median Time to Platelet Engraftment [ Time Frame: 1.5 years ]
    Determined for all participants who completed at least 75% of the planned doses. Platelet engraftment was as defined by the Center for International Blood and Marrow Transplant Research as the first of 3 consecutive days of a platelet count 20,000/mL without platelet trans-fusions for 7 days and/or the first day of a platelet count 100,000/mL without platelet transfusions for 7 days.
  • Median Number of Platelet Transfusions up to the Day of Engraftment [ Time Frame: baseline to day of engraftment ]


Original Secondary Outcome: to collect preliminary efficacy data for accelerating the recovery of megakaryopoiesis and meaningful platelet production by eltrombopag [ Time Frame: 1.5 years ]

Information By: University of Rochester

Dates:
Date Received: May 15, 2009
Date Started: December 2009
Date Completion:
Last Updated: April 1, 2016
Last Verified: April 2016