Open Label Study to Evaluate the Activity of Imetelstat in Patients With Essential Thrombocythemia or Polycythemia Vera (ET/PV)
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|ClinicalTrials.gov Identifier: NCT01243073|
Recruitment Status : Completed
First Posted : November 18, 2010
Last Update Posted : January 26, 2016
|Condition or disease||Intervention/treatment||Phase|
|Essential Thrombocythemia Polycythemia Vera||Drug: Imetelstat||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Study Start Date :||December 2010|
|Actual Primary Completion Date :||October 2013|
|Actual Study Completion Date :||April 2015|
Induction dosing of 9.4 mg/kg weekly, followed by intermittent maintenance dosing.
- 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.
- 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.
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 ClinicalTrials.gov identifier (NCT number): NCT01243073
|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, 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|