Open-label study of oral nilotinib in children with Ph+ CML or Ph+ ALL

• Research type

  Research Study

• Full title

  A multi-center, open-label, pharmacokinetic study of oral nilotinib in pediatric patients with Gleevec® (imatinib)- resistant/intolerant Ph+ CML chronic phase (CP) or accelerated phase (AP) or with refractory/relapsed Ph+ ALL

• IRAS ID

  63982

• Contact name

  Pamela Kearns

• Sponsor organisation

  Novartis Pharma Services AG

• Eudract number

  2010-018419-14

• Clinicaltrials.gov Identifier

  NCT01077544

• Research summary

  Philadelphia chromosome positive (Ph) leukemias, both chronic myelogenous leukemia (CML) and Ph acute lymphoblastic leukemia (ALL), are rare malignancies in childhood. They are mainly characterised by an abnormal chromosome called the ??Philadelphia chromosome? which creates an abnormal protein (BCRABL) hat causes leukemia cells to grow. Drugs that block the effects of this protein can be used to treat this type of leukaemia. The drug that will be tested in this study is called Nilotinib. Nilotinib has been developed to stop the abnormal protein (BCRABL) that causes leukemia cells to grow. In studies in adult patients, the ability to target this protein seems to be higher in nilotinib than in Gleevec©/Glivec©.This study will be conducted as a multicenter, open label study. The purpose is to learn how quickly the study drug nilotinib is absorbed and broken down by the body when given to children that have either Ph CML in chronic (CP) or accelerated phase (AP) that has not improved with a treatment called Gleevec©/Glivec© or, that have Ph ALL that has returned or has continued despite ongoing treatment. The data will be reviewed to understand the drug??s safety and to determine how well the drug works in children. Approximately 14 24 children worldwide will take part. Based on weight and height, they will receive a dose equal to the approved adult dose. The participant will be asked to come to the study site up to 18 times over 1 year. They may be followed for survival until the last patient completes the study. This study procedures include physical examination, blood samples, lumbar puncture, bone marrow biopsy or aspirate, echocardiogram, ECG. Ongoing safety monitoring will also be conducted by an IDMC. If at the Investigator's discretion, it is determined that the patient will benefit from treatment beyond 12 cycles, the patient will have the option to enroll into an extension study.

• REC name

  Yorkshire & The Humber - Sheffield Research Ethics Committee

• REC reference

  10/H1308/84

• Date of REC Opinion

  28 Mar 2011

• REC opinion

  Further Information Favourable Opinion