ARD12042- SAR302503 in patients with PV or ET
Research type
Research Study
Full title
ARD12042 - A Randomized Phase II, Open-Label study of the Efficacy and Safety of Orally Administered SAR302503 in patients with polycythemia vera (PV) or essential thrombocythemia (ET) who are resistant or intolerant to hydroxyurea.
IRAS ID
85273
Contact name
Claire Harrison
Sponsor organisation
sanofi aventis recherche et developpement
Eudract number
2011-001847-58
Clinicaltrials.gov Identifier
Research summary
Myelofibrosis (MF) is a chronic disorder where the bone marrow fails to produce normal blood cells. The bone marrow is replaced with collagen fibrosis which impairs the bone marrow's ability to generate new blood cells. As a consequence patients typically present with signs and symptoms caused by failure of the bone marrow (anemia, thrombocytopenia, leucopenia). MF can develop spontaneously on its own (primary MF) or following another myeloproliferative disease such as PV (post PV) or ET (post ET). Patients can also suffer debilitating constitutional symptoms, such as weight loss, fatigue, night sweats, itching and cough. In most cases, patients can have an enlarged spleen (splenomegaly) causing pain or fullness below the ribs. MF usually affects patients with advanced age but reports on young people do exist. Current available treatments for MF, such as blood transfusion, red blood cell production stimulating agents or hydroxycarbamide have not been shown to influence survival and are often used for palliative purposes only. Although bone marrow transplant can cure MF, this procedure carries a high mortality and morbidity rate and is precluded by age, poor performance status and co-morbidities. Several genetic mutations have been found in patients with MF. Janus kinase 2 (JAK2) mutations were described in approximately 50% of patients with PMF. Despite the precise contribution of these mutations not being currently defined, the hyperactivation of JAK-STAT due to mutations does lead to myeloproliferative syndrome-associated features, making JAK2 a reasonable target for treatment of this disease. SAR302503 is a protein kinase inhbitor, selective to JAK2, and is being developed as an orally available treatment to MF. This study proposes to investigate the safety and efficacy of SAR302503 on the reduction of spleen volume, overall survival, symptoms and quality of life in patients with PMF compared to placebo.
REC name
London - Brent Research Ethics Committee
REC reference
11/LO/1503
Date of REC Opinion
3 Oct 2011
REC opinion
Favourable Opinion