A UK Phase IIIB expanded access study of INC424 in myelofibrosis
Research type
Research Study
Full title
A UK open-label, multicentre, exploratory Phase II study of INC424 for patients with primary myelofibrosis (PMF) or post polycythemia myelofibrosis (PPV MF) or post-essential thrombocythaemia myelofibrosis (PET-MF)
IRAS ID
92004
Contact name
Adam Mead
Sponsor organisation
Novartis Pharmaceuticals UK Ltd.
Eudract number
2011-005066-38
ISRCTN Number
N/A
Clinicaltrials.gov Identifier
N/A
Research summary
Myelofibrosis is a bone marrow disorder characterised by abnormal scarring in the bone marrow associated with increase in spleen size and systemic symptoms such as weight loss, itching and night sweats. Myelofibrosis is characterised by abnormal growth factor signals which are mediated, in part, by proteins called JAKs which are frequently show abnormal levels of activity in patients with myelofibrosis, in many cases caused by direct damage to DNA encoding the JAK2 gene. INC424 is an inhibitor of JAK1 and JAK2 proteins that has shown considerable effectiveness as a treatment for myelofibrosis in Phase II & III clinical trials. The trials showed a decrease in the spleen size, along with improvements in symptoms associated with the disease such as weight loss, pruritus (itchiness) and fever. The purpose of this study is to collect further safety data, along with efficacy and Quality of Life data. It will also provide access to INC424 for patients with myelofibrosis, for whom there are very few effective treatments currently available. Patients will receive INC424 orally on a daily basis. Patients will visit the clinic every 4 weeks up to week 12 and then every 12 week s for physical exams, ECGs, questionnaires and blood testing until the end of the study (15th June 2013). This is a UK study and approximately 33 patients will take part.
REC name
South Central - Oxford C Research Ethics Committee
REC reference
11/SC/0499
Date of REC Opinion
16 Jan 2012
REC opinion
Further Information Favourable Opinion