Phase II Ponatinib in Refractory CML and Ph+ALL Version 2.0 27Oct2010

• Research type

  Research Study

• Full title

  A Pivotal Phase 2 Trial of Ponatinib (AP24534) in Patients with Refractory Chronic Myeloid Leukemia and Ph+ Acute Lymphoblastic Leukemia

• IRAS ID

  63420

• Contact name

  Jane Apperley

• Sponsor organisation

  Ariad Pharmaceuticals

• Eudract number

  2010 - 020414 - 28

• ISRCTN Number

  N/A

• Clinicaltrials.gov Identifier

  NCT01207440

• Research summary

  This is a clinical trial to establish whether a new drug, ponatinib, is effective in patients with chronic myeloid leukaemia (CML) and Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph ALL). Ponatinib is an experimental anti-cancer agent. Laboratory studies and animal research suggest that ponatinib stops cancer cells from growing by inhibiting different kinds of proteins in cancer cells. In particular, laboratory studies suggest that ponatinib may be effective in inhibiting a protein called Bcr-Abl which is the causative oncogene in CML and Ph ALL. There is also evidence that ponatinib may be able to stop an altered (mutated) form of Bcr-Abl known as the T315I mutation, for which there are currently no drugs available. Patients participating in this study will have CML or Ph ALL for which effective forms of treatment (such as chemotherapy or other anti-cancer treatments) have failed to control the growth of the leukaemia. This is a multi-centre, international study. The duration will be up to 25 months. Each patient will undergo a period of up to 3 weeks for screening and then begin once daily dosing with ponatinib for up to 24 months. Throughout the study, the investigator will monitor the patient’s condition by examining them and performing various blood tests and bone marrow examinations. Most of the tests are similar to those performed as part of the standard care of patients with leukaemia. Some of the tests are done to find out as early as possible if ponatinib has side effects.

• REC name

  London - Bloomsbury Research Ethics Committee

• REC reference

  10/H0713/88

• Date of REC Opinion

  21 Feb 2011

• REC opinion

  Further Information Favourable Opinion