A Phase 1B study of INC424 in myelofibrosis with thrombocytopaenia

• Research type

  Research Study

• Full title

  A Phase 1b, open-label, dose-finding study of JAK inhibitor INC424 tablets administered orally to patients with Primary Myelofibrosis (PMF), Post-Polycythemia Vera-Myelofibrosis (PPV-MF) or Post-Essential Thrombocythemia-Myelofibrosis (PET-MF) and baseline platelet counts more than/= 50 x 10 9/L and less than 100 x 10 9/L

• IRAS ID

  74662

• Contact name

  Claire Harrison

• Sponsor organisation

  Novartis Pharma AG

• Eudract number

  2010-023055-29

• ISRCTN Number

  n/a

• Clinicaltrials.gov Identifier

  n/a

• Research summary

  This study, INC424A2201, is designed to establish the safe starting dose of INC424 in patients with myelofibrosis and a low platelet count (stratum 1: 75-99 x 10 9/L and stratum 2 50-74 x 10 9/L). Patients who give their informed consent will be interviewed and examined by the study doctor and have blood samples collected. Patients that meet the entry criteria will attend the first clinic visit, at which they will be interviewed and examined by the study doctor, will have an electrocardiogram (ECG), complete a questionnaire, have weight and vital signs determined and blood samples collected. These assessments and procedures will be repeated at some or all of the subsequent clinic visits. Patients will also be instructed on how to use the electronic hand-held device to score their symptoms each evening. All patients will receive INC424 tablets, but the size of the dose will vary between patients. The first group per stratum (3-6 patients) will receive 10 mg/day (5 mg twice-a-day) and, if this is safe, the next group will receive 15 mg/day. The starting dose will be increased step-wise in 5 mg/day steps until the maximum safe starting dose is determined or the 30 mg/day dose is achieved. The maximum safe starting dose will then be studied further in the dose expansion phase of the trial in a further 20 patients (10 per stratum).Over the first 4 weeks of treatment, safety monitoring is intensive, with a clinic visit and a blood-sampling visit (2 visits) each week for 4 weeks, then fortnightly for the next 4 weeks. Thereafter, the monitoring is monthly for four months, unless the INC424 dose is increased, in which case intensive monitoring will be resumed. At the end of the study patients benefiting from INC424 will continue INC424 in an extension study.

• REC name

  South Central - Berkshire Research Ethics Committee

• REC reference

  11/SC/0059

• Date of REC Opinion

  27 Apr 2011

• REC opinion

  Further Information Favourable Opinion