A Randomised Phase III Study of Elacytarabine vs. Investigator's Choice in Patients With Late Stage Acute Myeloid Leukaemia
The study investigates the new nucleoside analogue derivative, elacytarabine, as treatment
for patients with relapsed or refractory Acute Myeloid Leukemia (AML). To be included in the
study, patients must have failed to respond to two or three different therapies for AML, or
have obtained remission but then relapsed within a relatively short period of time. Patients
of age ≥ 65 with adverse cytogenetics can be included in the study after having received
one and up to three previous induction/re-induction therapies.
Elacytarabine is an investigational drug which is not commercially available. It is the
elaidic acid ester derivative of cytarabine. Cytarabine is routinely used in the treatment
of patients with AML. A substantial portion of AML patients have a deficient uptake of
cytarabine, often explained by lack of a transport protein (hENT1) in the leukemic cell
membrane. Due to the elaidic acid (a naturally occurring fatty acid), cellular uptake of
elacytarabine is independent of this transport protein.
Patients included in the study will be randomized to elacytarabine or control treatment.
Since there is no standard therapy for relapsed or refractory AML, there is a list of 7
control treatments and the investigator has to choose one that is locked before
randomization.
Elacytarabine is given as a continuous infusion over five days, followed by a rest period of
minimum two weeks. Investigator's choice treatment is given according to the specific
routine.
After each course response evaluation and a decision on further treatment will be made.
Repeated courses of elacytarabine and control treatment might be needed to attain and/or
maintain complete remission or clinical benefit.
After the end of study treatment, all patients will be followed for relapse and survival.
Interventional
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Overall survival
Time from date of randomisation until the date of death
Until 300 events occur
No
David Rizzieri, MD
Principal Investigator
Duke University Medical Center, Durham, NC, USA
United States: Food and Drug Administration
CP4055-306
NCT01147939
June 2010
June 2013
Name | Location |
---|---|
New York Medical College | Valhalla, New York 10595 |
Western Pennsylvania Hospital | Pittsburgh, Pennsylvania 15224 |
Rush University Medical Center | Chicago, Illinois 60612-3824 |
USC/Norris Comprehensive Cancer Center and Hospital | Los Angeles, California 90033-0804 |
Duke University Medical Center | Durham, North Carolina 27710 |
Northwestern University | Chicago, Illinois 60611 |
Memorial Sloan-Kettering | New York, New York |
Northern New Jersey Cancer Associates | Hackensack, New Jersey 07601 |
Sarah Cannon Research Institute | Nashville, Tennessee 37203 |
Scripps Cancer Center Clinical Research | San Diego, California 92121 |
The Jewish Hospital | Cincinnati, Ohio 45236 |
St. Francis Hospital and Health Center | Beech Grove, Indiana 46107 |
The Blood and Marrow Transplant Group of GA | Atlanta, Georgia 30342 |
St. Francis Hospital | Greenville, South Carolina 29601 |
UCLA School of Medicine, Division of Hematology/Oncology | Los Angeles, California 90095 |
Rocky Mountain Blood and Bone Marrow Transplant Program | Denver, Colorado 80218 |
Shands at the University of Florida | Gainesville, Florida 32610 |
Winship Cancer Institute at Emory | Atlanta, Georgia 30322 |
University of Iowa Hopsitals | Iowa City, Iowa 52242 |
LSU Health Sciences Center, | Shreveport, Louisiana 71103 |
New York Presbyterian Hospital, Weill-Cornell Medical College | New York, New York 10021 |
Wake Forest University, Health Sciences Section on Hematology and Oncology | Winston-Salem, North Carolina 27157-1082 |
Froedtert Hospital, Medical College of Wisconsin | Milwaukee, Wisconsin 53226 |