ARD12181: SAR302503 in MF patients previously treated with Ruxolitinib

• Research type

  Research Study

• Full title

  A Phase II, Multicenter, Open Label, Single Arm Study of SAR302503 in Subjects Previously Treated with Ruxolitinib and with a Current Diagnosis of Intermediate-2 or High-Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis

• IRAS ID

  96129

• Contact name

  Claire Harrison

• Sponsor organisation

  sanofi-aventis recherche et developpement

• Eudract number

  2011-005226-21

• ISRCTN Number

  n/a

• 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 (anaemia, thrombocytopenia, leucopenia). MF can develop spontaneously on its own 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 causing pain or fullness below the ribs. MF usually affects patients with advanced age but reports on young people do exist. Janus kinase 2 (JAK2) mutations were described in approximately 50% of subjects with PMF. It is also expressed in 95% of the PV subjects and in 50% of essential thrombocythemia (ET) subjects. The contribution of these mutations is currently not yet understood, but it has been shown that JAK2 plays a major role in the bone marrow's overproduction of red bloods cells and platelets in PV & ET, making JAK2 a reasonable target for treatment of MPDs. SAR302503 is a protein kinase inhibitor that targets JAK2, it is being developed as an oral capsule for the treatment of MF. Another JAK inhibitor currently leading is ruxolitinib, however if platelet counts are below <50 x 109/L, dosing has to be stopped. This indicates some subjects need other options to manage their disease-related symptoms or to improve their response to treatment. This study proposes to investigate the efficacy & safety of SAR302503 when administered to patients with MF previously treated with Ruxolitinib.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  12/LO/0231

• Date of REC Opinion

  12 Mar 2012

• REC opinion

  Further Information Favourable Opinion