Extension Study Evaluating the Long Term Safety and Efficacy Study of CYT387 in Primary Myelofibrosis (PMF) or Post-polycythemia Vera (PV) or Post-essential Thrombocythemia (ET)
This extension protocol to the core study CCL09101 allows patients who have tolerated the drug and derived a clinical benefit, to continue to receive treatment beyond the 9 cycles of the core protocol. Long term safety and efficacy of CYT387 will be evaluated.
Post-Polycythemia Vera Myelofibrosis
Post-Essential Thrombocythemia Myelofibrosis
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II, Open-Label Extension Study Evaluating the Long Term Safety, Tolerability & Efficacy of Orally-Administered CYT387 in Primary Myelofibrosis or Post-Polycythemia Vera or Post-Essential Thrombocythemia Myelofibrosis|
- To determine the long term safety and tolerability of orally-administered CYT387 in patients with PMF or post-ET/PV MF following completion of core study CCL09101 [ Time Frame: Safety monitoring will be undertaken for all patients every 3 months ] [ Designated as safety issue: No ]
- To obtain information on the long term effectiveness of orally-administered CYT387 in patients with PMF or post-ET/PV MF [ Time Frame: Every three months ] [ Designated as safety issue: No ]Measured by complete response (CR) rate, partial response (PR) rate and clinical improvement (CI) rate according to IWG-MRT consensus criteria
|Study Start Date:||November 2010|
|Estimated Study Completion Date:||June 2014|
|Estimated Primary Completion Date:||June 2014 (Final data collection date for primary outcome measure)|
Patients will continue receiving the same doses as assigned during the CCL09101 protocol; up to 400 mg once per day (QD). CYT387 will be administered orally as a single daily dose (at least 20 and no more than 28 hours apart, preferably in a fasted state at least two hours before and one hour after a meal), except for patients on the twice daily (BID) dosing regime (150 mg BID).
The myeloproliferative neoplasms (MPN), most notably polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF) are a diverse but inter-related suite of clonal disorders of pluripotent hematopoietic stem cells (Tefferi et al., 2008). The MPN share a range of biological, pathological, and clinical features including the relative overproduction of one or more cells of myeloid origin, growth factor independent colony formation in vitro, marrow hypercellularity, extramedullary hematopoiesis, spleno- and hepatomegaly, and thrombotic and/or hemorrhagic diatheses (Tefferi et al., 2005).
This is a multi centre, open-label, extension study of the core study (CCL09101). The primary aims of the study will be to determine the long term safety and tolerability of orally-administered CYT387 when administered as a capsule dose, on a 28-day treatment cycle.
Following completion of the core study (CCL09101), patients who have tolerated the drug and derived clinical benefit will continue to be treated with CYT387 administered orally.
Subjects will be evaluated every three months for up to 24 cycles of CYT387 treatment. Subjects will return for a follow-up visit 30 days after completion of the last dose of study drug.
|United States, California|
|Stanford Cancer Center|
|Stanford, California, United States, 94305-5821|
|United States, Massachusetts|
|Dana-Farber Cancer Institute|
|Boston, Massachusetts, United States, 02115|
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|The Royal Melbourne Hospital|
|Parkville, Victoria, Australia, 3050|
|Princess Margaret Hospital|
|Toronto, Ontario, Canada, M5G 2M9|
|Jewish General Hospital|
|Montreal, Quebec, Canada, H3T 1E2|
|Study Chair:||Ayalew Tefferi, MD||Mayo Clinic|