Phase 2 Study of SAR302503 in Patients With Myelofibrosis
Status:
Completed
Trial end date:
2014-04-01
Target enrollment:
Participant gender:
Summary
Primary Objective:
- To evaluate the efficacy of daily oral doses of 300 mg, 400 mg, and 500 mg SAR302503 for
the reduction of spleen volume as determined by magnetic resonance imaging (MRI).
Secondary Objectives:
- To evaluate the safety of SAR302503.
- To evaluate the pharmacokinetics (PK) of SAR302503 after single and repeat doses.
- To evaluate the pharmacodynamics of SAR302503 as measured by changes in JAK2V617F allele
burden in those patients with JAK2V617F mutation, changes in substrate phosphorylation
in the JAK-STAT signal transduction pathway, and the expression of cytokines.
- To measure improvement in baseline Myeloproliferative Neoplasm (MPN) associated
symptoms, as well as overall impact in quality of life (QOL), through serial
administration of the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF).
- To measure generic health-related quality of life (HRQL) and utility values using the
EQ-5D questionnaire.