Clinical Trial: Open Label Study to Evaluate the Activity of Imetelstat in Patients With Essential Thrombocythemia or Polycythemia Vera

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

Official Title: A Phase II Trial to Evaluate the Activity of Imetelstat (GRN163L) in Patients With Essential Thrombocythemia or Polycythemia Vera Who Require Cytoreduction and Have Failed

Brief Summary: This is a phase II open-label study of single agent imetelstat in patients with essential thrombocytopenia or with polycythemia vera who have failed or are intolerant to at least one prior therapy, or who refuse standard therapy.

Detailed Summary:

For patients with ET: To obtain a preliminary estimate of efficacy of imetelstat, as measured by best hematologic response within the first year of therapy in patients with ET who have failed or are intolerant to at least one prior therapy, or who have refused standard therapy.

For patients with PV: To obtain a preliminary estimate of efficacy of imetelstat, as measured by maintenance of Hct < 45% in men and < 42% in women (or pre-specified Hct count that is tolerable) without phlebotomy or myelosuppressive therapy within the first year of therapy in patients with PV who have failed or are intolerant to at least one prior therapy, or who have refused standard therapy.


Sponsor: Geron Corporation

Current Primary Outcome: Hematologic Response [ Time Frame: From time of first dose (cycle 1 day 1) through end of study (12 mos after last participant is dosed) ]

Primary objectives are as follows: ET patients - best hematologic response within the first year of therapy and PV patients - maintenance of Hct < 45% in men and < 42% in women (or pre-specified Hct count that is tolerable) without phlebotomy or myelosuppressive therapy within the first year of therapy. Secondary objectives, to determine the durability of hematologic response and to determine the rate of phlebotomy required within the first year of therapy.


Original Primary Outcome: Hematologic Response [ Time Frame: From time of first dose (cycle 1 day 1) through end of study (12 mos after last participant is dosed) ]

The primary objective of this study is to obtain a preliminary estimate of efficacy of imetelstat, as measured by hematologic response - defined by the Modified European Leukemianet Consensus Conference Response Criteria, in patients with essential thrombocythemia (ET) who have failed or are intolerant to at least one prior therapy, or who have refused standard therapy. Secondly, the study will evaluate the durability of the hematologic response.


Current Secondary Outcome: Safety and tolerability: Number of Patients with Hematological Toxicities, Non-Heme Grade 3 and 4 AEs, and Hemorrhagic Events [ Time Frame: From time of first dose (cycle 1 day 1) through end of study (12 mos after last paricipant is dosed) ]

The safety and tolerability of imtelstat will be assessed by the incidence, nature, relatedness and severity of adverse events, laboratory abnormalities and vital signs.


Original Secondary Outcome: Same as current

Information By: Geron Corporation

Dates:
Date Received: November 11, 2010
Date Started: December 2010
Date Completion:
Last Updated: December 22, 2015
Last Verified: December 2015