Open Label Study to Evaluate the Activity of Imetelstat in Patients With Essential Thrombocythemia or Polycythemia Vera (ET/PV)
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.
Drug: Standard of Care
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|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 or Are Intolerant to Previous Therapy, or Who Refuse Standard Therapy|
- Hematologic Response [ Time Frame: From time of first dose (cycle 1 day 1) through end of study (12 mos after last participant is dosed) ] [ Designated as safety issue: No ]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.
- 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) ] [ Designated as safety issue: Yes ]The safety and tolerability of imtelstat will be assessed by the incidence, nature, relatedness and severity of adverse events, laboratory abnormalities and vital signs.
|Study Start Date:||December 2010|
|Estimated Study Completion Date:||January 2016|
|Estimated Primary Completion Date:||January 2014 (Final data collection date for primary outcome measure)|
Induction dosing of 9.4 mg/kg weekly, followed by intermittent maintenance dosing.
Drug: Standard of Care
Standard of care
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.
|United States, California|
|City of Hope|
|Duarte, California, United States, 91010|
|United States, Florida|
|MDACC - Orlando|
|Orlando, Florida, United States, 32806|
|United States, Illinois|
|University of Chicago|
|Chicago, Illinois, United States, 60637|
|United States, Maryland|
|Johns Hopkins University - Bunting Blaustein Cancer Research Building|
|Baltimore, Maryland, United States, 21205|
|United States, New York|
|Mount Sinai School of Medicine - Tisch Cancer Institute|
|New York, New York, United States, 10029|
|United States, South Carolina|
|Saint Francis Hospital|
|Greenville, South Carolina, United States, 29601|
|United States, Texas|
|MD Anderson Cancer Center|
|Houston, Texas, United States, 77030|
|University Hospital of Essen - West German Cancer Center|
|Medizinische Klinik II, Abt. Hämatologie und Onkologie - Johann Wolfgang Goethe Universität|
|Frankfurt, Germany, D-60590|
|Hematology Oncology Center - Ludwig-Maximilians, University Munich Medical School|
|Munich, Germany, 80331|
|University Hospital Regensburg - Uniklinik Regensburg|
|INSELSPITAL, University Hospital Bern|
|Bern, Switzerland, CH - 3010|
|Study Director:||Stephen Kelsey, MD FRCP FRCPath||Geron Corporation|