Research protocols

CML protocols research


Title of study : A Randomized, Open-label, Phase 2 Trial of Ponatinib in Patients With Resistant Chronic Phase Chronic Myeloid Leukemia to Characterize the Efficacy and Safety of a Range of Doses (OPTIC)

Participating center : Jewish General Hospital, PI: Dre. Sarit Assouline

Abstract  : The clinical trial OPTIC (Optimizing Ponatinib Treatment In CML; trial number on NCT02467270) is an international phase 2 study enrolling patients with CP-CML who are resistant to at least two approved TKIs. These patients are randomized equally to receive once-daily administration of 45 mg (cohort A), 30 mg (cohort B) or 15 mg (cohort C) of ponatinib. Patients in cohorts A and B will have their daily dose reduced to 15 mg upon achievement of major cytogenetic response (MCyR). The primary endpoint of the trial is MCyR by 12 months for each cohort. Secondary endpoints include rate of vascular occlusive events in each dose cohort, rates of adverse events and rates of serious adverse events. Other secondary endpoints include cytogenetic, molecular and hematologic response rates, tolerability, duration of response, time to response, disease control rate, progression-free survival and overall survival. Preliminary data from the OPTIC trial is expected at the end of 2016.


Title of study : Treatment-free Remission Accomplished With Dasatinib in Patients With CML

Participating center : Hôpital Maisonneuve-Rosemont, PI : Dr. Lambert Busque ; Hôpital de l’Enfant-Jésus, PI : Dr. Robert Lepage

Abstract : The purpose of this study (NCT02268370) is to find out how to increase the potential for achieving an “operational cure” from chronic myeloid leukemia. An “operational cure” is a state in which a person does not require further treatment, although there may be some remaining cancer cells. Patients would normally remain on a TK inhibitor indefinitely within a standard of care setting for chronic myeloid leukemia. Within this clinical trial, patients will discontinue their TK inhibitor prematurely. If any signs of progression are identified, dasatinib will be introduced. This research is being done because dasatinib has been shown to achieve a greater response in a much higher proportion of patients as compared to imatinib. Dasatinib is approximately 300 times more potent than imatinib, and it is possible that a greater response can be achieved by dasatinib than by imatinib.


Title of study : An Open-label Bosutinib Treatment Extension Study For Subjects With Chronic Myeloid Leukemia (CML) Who Have Previously Participated In Bosutinib Studies B1871006 Or B1871008

Participating center : Jewish General Hospital, PI: Dre. Sarit Assouline

Abstract : The objective of the study (NCT01903733) is to provide long term access to bosutinib treatment and assess long term safety, tolerability and duration of clinical benefit, without any formal hypothesis testing; therefore, there is no formal primary endpoint.


NPM protocol research

Title of study : A Phase Ib, Multi-center, Open-label, Dose-escalation Study of PIM447 in Combination With Ruxolitinib (INC424) and LEE011 Administered Orally in Patients With Myelofibrosis

Participating center : Jewish General Hospital, PI: Dre Shireen Sirhen

Abstract : This is a phase Ib study (NCT02370706) with the primary purpose is to estimate the MTD and/or RDE for the triple combination of PIM447, formerly LGH447, plus ruxolitinib and LEE011 as well as for the doublets, PIM447 plus ruxolitinib, and LEE011 plus ruxolitinib, in patients with myelofibrosis (MF). Each regimen will be assessed for safety, tolerability, pharmacokinetics (PK) and pharmacodynamic effects, and preliminary anti-myelofibrosis activity, including changes in spleen volume, JAK2V617F allele burden, and hematologic response.


For research projects initiated by the pharmaceutical industry see sections: research protocol in CML or research protocol in MPN