Phase I/II study of novel ruxolitinib combinations in MF Patients
Research type
Research Study
Full title
A randomized, open-label, phase I/II open platform study evaluating safety and efficacy of novel ruxolitinib combinations in myelofibrosis patients.
IRAS ID
263252
Contact name
Claire Harrison
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2019-000373-23
Duration of Study in the UK
4 years, 6 months, 5 days
Research summary
Myelofibrosis (MF) is defined by progressive bone marrow (BM) fibrosis and a consecutive reduction of blood cells. The disruption of the medullary erythropoietic niche is the primary mechanism governing the bone marrow failure and anemia, which typify MF. Nearly 40% of MF patients have hemoglobin (Hb) levels < 10 g/dL at diagnosis. Furthermore, anemia is the disease feature most consistently associated with poor prognosis in MF. Ruxolitinib demonstrates improvements in splenomegaly and constitutional
symptoms, however, does not improve anemia.
The purpose of this study is to investigate the safety, pharmacokinetics (PK) and preliminary efficacy of combinations treatment of ruxolitinib with 3 novel compounds: siremadlin, crizanlizumab and MBG453 in MF subjects. These combination therapies may deliver transformational clinical benefits such as improvement of progression free survival (PFS) as a consequence of superior disease control or reduction of the malignant clone, associated with an improvement of cytopenia and in particular anemia, as well as improvement in quality of life (QoL) as captured by relevant patient reported outcomes measurements (PROs).REC name
Wales REC 1
REC reference
19/WA/0243
Date of REC Opinion
8 Oct 2019
REC opinion
Further Information Favourable Opinion