AC220 in patients with Acute Myeloid Leukaemia

• Research type

  Research Study

• Full title

  A PHASE 2 OPEN-LABEL, AC220 MONOTHERAPY EFFICACY (ACE) STUDY IN PATIENTS WITH ACUTE MYELOID LEUKEMIA (AML) WITH FLT3-ITD ACTIVATING MUTATIONS

• IRAS ID

  31029

• Contact name

  Alan K Burnett

• Sponsor organisation

  Ambit Biosciences Corporation

• Eudract number

  2009-013093-41

• ISRCTN Number

  not available

• Research summary

  This study is investigating a new treatment for Acute Myeloid Leukaemia (AML), which is a fast-growing cancer of the blood and bone marrow. Without effective treatment, AML will rapidly cause death. Chromosome analysis (cytogenetics) is now of considerable importance in the diagnosis and treatment planning of AML. Detailed examination of chromosomes from leukaemic cells shows distinctive abnormalities in almost all cases. Recent studies have shown that 15-25% of AML patients have an abnormality of a gene called FLT3-ITD in their leukaemia cells. The prognosis for patients with the FLT3-ITD gene abnormality is significantly worse than that of patients without the abnormality when treated with standard therapy.The study drug, AC220, is a new agent that blocks certain receptors (tyrosine kinase) on the leukaemia cells that are responsible for cell growth and proliferation. The primary objective of this Phase 2 study is to establish if AC220 is an effective treatment for AML in participants who have the FLT3-ITD gene abnormality in their leukaemia cells and have failed to respond to standard therapy (i.e. refractory or relapse). Approximately 180 participants (169 evaluable) are planned to be enrolled into the study. Every participant will receive the same dose of AC220, 200mg given daily in a 28-day treatment cycle. There will be no breaks between the cycles - continuous monotherapy. Participants will be assessed regularly to determine if AC220 is safe and can be tolerated as a potential treatment for AML. After three full treatment cycles participants will be assessed for complete or partial remission to treatment. Participants who respond will continue on AC220 as post-remission and maintenance therapy. Those participants who stop treatment with AC220 will be followed up every three months to document initiation of new treatments and overall survival.The study is expected to last 2 years.

• REC name

  Wales REC 3

• REC reference

  09/MRE09/60

• Date of REC Opinion

  15 Dec 2009

• REC opinion

  Favourable Opinion