The purpose of this study is to assess the safety, efficacy, pharmacokinetics (PK),
pharmacodynamics (PD), and immunogenicity of OMS721 in patients with thrombotic
microangiopathies (TMA).
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT02222545.
This is a Phase 2, uncontrolled, 3-stage, ascending-dose-escalation study in patients
with 1 of 3 forms of TMA: atypical hemolytic uremic syndrome (aHUS), thrombotic
thrombocytopenia (TTP), and hematopoietic stem cell transplant - associated TMA
(HSCT-associated TMA). In Stage 1 of the study, OMS721 was administered to 3 cohorts,
with dose escalation by cohort to identify the optimal dosing regimen. In Stage 2, the
dose selected in the first stage was administered to expanded cohorts of patients with
distinct etiologies (aHUS alone in 1 cohort and TTP or HSCT-TMA in the other cohort).
Patients completing Stage 2 were eligible for continued treatment in Stage 3 if they
tolerated OMS721 treatment and derived clinical benefit. Enrollment in the study has been
completed.
Lead OrganizationOmeros Corporation
