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  • A Phase 2, Uncontrolled, Three-Stage, Dose-Escalation Cohort Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, Immunogenicity, and Clinical Activity of OMS721 in Adults With Thrombotic Microangiopathies Rochester, Minn.

    This is a Phase 2, uncontrolled, three-stage, ascending-dose-escalation study in subjects with one of three forms of TMA: atypical hemolytic uremic syndrome (aHUS), thrombotic thrombocytopenic purpura (TTP), or hematopoietic stem cell transplant (HSCT)-associated TMA. In the first stage, OMS721 will be administered to escalating dose cohorts of three subjects per cohort to identify the optimal dosing regimen. After enrollment and treatment of each cohort there will be a safety review to determine whether dose escalation should proceed. In the second stage, the dose selected in the first stage will be administered to expanded cohorts of 40 subjects per cohort with distinct etiologies (aHUS alone in one cohort and TTP or HSCT-TMA in the other cohort). Subjects completing the second stage may be eligible for continued treatment in the third stage if they have tolerated OMS721 treatment and derived clinical benefit. OMS721 is to be used in conjunction with standard of care treatments. Standard of care treatments are not to be delayed or withheld from subjects entering this study. These treatments should be initiated according to local standard of care. For example, plasma exchange should not be delayed or withheld from subjects suspected of having TTP while waiting for confirmatory ADAMTS13 laboratory results and potential initiation of OMS721 treatment. Subjects who have failed a treatment, e.g., subjects with plasma-therapy resistant aHUS, do not require continued treatment with the failed therapy.