ETB115E2201: A phase II, open-label, non-controlled, intra-patient dose-escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in pediatric patients with refractory, relapsed or treatment naïve Severe Aplastic Anemia (SAA) or recurrent Aplastic Anemia (AA)
Study ID: STU 052017-089
The purpose of this study is to characterize the PK of eltrombopag at steady state after oral administration, in patients with previously untreated Saa (Cohort B) or who have refractory/relapsed Saa, or who have recurrent aa after treatment with iST for Saa (Cohort a).
Primary objective: * To characterize the PK of eltrombopag at steady state after oral administration in pediatric patients with Saa. Key Secondary objectives: * To determine the safety and tolerability of eltrombopag given orally in pediatric patients with Saa. * To assess the efficacy of eltrombopag defined as overall response rate (oRR)