Phase III study of Ruxolitinib in Early Myelofibrosis
Research type
Research Study
Full title
A randomized, double-blind, placebo-controlled, multi-center, Phase III study investigating the efficacy and safety of ruxolitinib in Early Myelofibrosis patients with high molecular risk mutations
IRAS ID
194656
Contact name
Tim Somervaille
Contact email
Sponsor organisation
Novartis Pharmaceuticals UK Ltd
Eudract number
2014-004928-21
Clinicaltrials.gov Identifier
Duration of Study in the UK
4 years, 10 months, 21 days
Research summary
This study is a Phase III, double-blind, placebo-controlled study investigating the efficacy and safety of ruxolitinib in early myelofibrosis patients with high molecular risk mutations.
There are around 24 genes associated with myelofibrosis (MF) that will be analysed in the pre-screening part of the study to pre-identify patients who will benefit most from being in the study. Five of these genes are thought to be directly involved in the progression of MF and patients will need to have a change in one of these 5 genes to be eligible. These changes are regarded as High Molecular Risk Mutations (HMR).
Once eligibility has been confirmed, patients will enter the study. The study has two treatment arms (ruxolitinib and placebo) and consists of a screening period and, treatment parts 1 and 2.
Treatment Part 1: patients may receive ruxolitinib or placebo and be asked to take tablets two times per day until such time they do not benefit from the treatment. If a patients disease worsens due to spleen measurements or symptoms, patients may then receive ruxolitinib as per the dosage instructions for the already approved medicine. This second part of the study is called Treatment period 2.
Ruxolitinib is already approved in the USA for the treatment of intermediate or high-risk MF. In Europe ruxolitinib was approved by the EMA in August 2012 and is used for the treatment of disease-realted splenomegaly (enlarged spleen) or symptoms in patients with MF. Currently there are no approved treatments for patients with early MF who are also positive for the HMR gene mutations. This study will try to determine if the drug can delay HMR+ early stage MF disease from becoming more advanced and improve symptoms and survival of patients.
Study duration is approximately 48 weeks, however patients may continue on treatment for as long as they receive benefit.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
16/NW/0187
Date of REC Opinion
18 Apr 2016
REC opinion
Further Information Favourable Opinion