Clinical Research Directory

Inclusion Criteria: * Able to provide a signed and dated written informed consent prior to any study-specific procedures, sampling, or data collection. * A clinical diagnosis of HHT as defined by the Curaçao criteria * Baseline (1-month) ESS ≥ 4 * ECOG ≤ 2 * Anemia or parental iron infusion of at least 500 mg or transfusion of at least 2 units of RBCs within the preceding 24 weeks. * Adequate bone marrow function * Adequate renal function * Adequate hepatic function Exclusion Criteria: * Prior nonresponse or loss of response to an agent that inhibits AKT1 and/or AKT2 as the primary mechanism of action. * Diagnosis of DM requiring insulin treatment * Known significant bleeding sources other than nasal, GI, or menstrual/ uterine * Known underlying hypoproliferative anemia or clinically significant hemolytic anemia