NCT01147939

Study of Elacytarabine Versus Investigator's Choice in Patients With Late Stage Acute Myeloid Leukaemia (AML)

A Randomised Phase III Study of Elacytarabine vs. Investigator's Choice in Patients With Late Stage Acute Myeloid Leukaemia

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 381 (actual)
• Sponsor: Clavis Pharma · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to assess the efficacy and safety of elacytarabine versus investigator's choice treatment in patients with relapsed or refractory acute myeloid leukemia (AML).

Detailed description

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.

Conditions

• Acute Myeloid Leukemia (AML)

Interventions

Type	Name	Description
DRUG	Elacytarabine	Elacytarabine 2000 mg/m2/d administered as a continuous intravenous infusion (CIV) in a d 1-5 q3w cycle.
DRUG	Investigator's Choice	E.g. cytarabine single agent/combinations, hypomethylating agents, best supportive care (BSC)

Timeline

• Start date: 2010-06-01
• Primary completion: 2013-02-01
• Completion: 2013-06-01
• First posted: 2010-06-22
• Last updated: 2013-09-27

Locations

73 sites across 13 countries: United States, Australia, Belgium, Canada, France, Germany, Ireland, Italy, Norway, Poland, Romania, Spain, United Kingdom