A Phase 3 Study to Evaluate the Safety and Efficacy of OMS721 for the Treatment of Atypical Hemolytic Uremic Syndrome (aHUS) in Adults and Adolescents
The purpose of this study is to evaluate the platelet count change from baseline and safety of OMS721 (narsoplimab) in adults and adolescents with atypical hemolytic uremic syndrome (aHUS). The study will also evaluate pharmacokinetics (PK), pharmacodynamics (PD), and anti-drug antibody response (ADA).
• Are age \>= 12 years old at screening (Visit 1).
• Have a primary aHUS, diagnosed clinically, and have ADAMTS13 activity \> 5% in plasma. Participants are eligible with or without a documented complement mutation or anti-CFH antibody. Participants are categorized according to their response to plasma therapy (plasma exchange or plasma infusion):
‣ Plasma therapy-resistant aHUS participants must have all of the following:
• Screening platelet count \< 150,000/μL despite at least four plasma therapy treatments in a 7-day period prior to screening
∙ Evidence of microangiopathic hemolysis (at least one of:
∙ presence of schistocytes,
‣ serum LDH \> 1.5 times upper limit of normal (ULN), and
‣ haptoglobin \< LLN)
∙ Serum creatinine \> ULN
⁃ Plasma therapy-responsive aHUS participants must have all of the following:
• Have a documented history of requiring plasma therapy to prevent aHUS exacerbation defined as all of the following:
‣ decrease in platelet count \> 25% when plasma therapy frequency has been decreased (including discontinuation of plasma therapy)
⁃ LDH \> 1.5 times ULN when plasma therapy frequency has been decreased (including discontinuation of plasma therapy)
⁃ Have received plasma therapy at least once every 2 weeks at an unchanged frequency for at least 8 weeks before first dose of OMS721
• If sexually active and of childbearing potential, must agree to practice a highly effective method of birth control until the end of the study, defined as one that results in a low failure rate (i.e., less than 1% per year) when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine devices, sexual abstinence or vasectomized partner.
• Do not have access to eculizumab treatment, have not derived therapeutic benefit from eculizumab treatment, or have not been able to tolerate eculizumab treatment.