Safety and Efficacy Study of OMS721 in Patients With Atypical Hemolytic Uremic Syndrome

Launched by OMEROS CORPORATION · Jun 28, 2017

Keywords

ClinConnect Summary

This clinical trial is testing a new treatment called OMS721 for patients with atypical hemolytic uremic syndrome (aHUS), a rare condition that can affect the blood and kidneys. The main goals of the study are to see how well OMS721 works in increasing platelet counts and to check for any possible side effects. Researchers will also look at how the body processes the medication and whether the body develops any antibodies against it.

To be eligible for the trial, participants must be at least 12 years old and have a confirmed diagnosis of aHUS. They should also have specific blood test results that indicate their condition. Some people, like those with certain types of kidney disease or those who have had recent serious health issues, may not be eligible. If you or someone you know joins the study, you can expect to receive OMS721 and regular monitoring to track health changes and any side effects. This study is currently recruiting participants, and it aims to provide valuable information about a potentially new treatment for aHUS.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Competent to provide informed consent, or if a minor, have at least one parent or legal guardian to provide informed consent with written assent from the subject.
• • Are at least 12 years old at screening (Visit 1).
• • Have a clinically diagnosis of primary atypical hemolytic uremic syndrome (aHUS), with ADAMTS13 activity greater than 5% in plasma.
• • Plasma therapy-resistant aHUS patients must have a screening platelet count less than 150,000/uL, evidence of microangiopathic hemolysis, and serum creatinine greater than upper limit of normal.
• • Plasma therapy-responsive aHUS patients must have documented history of requiring plasma therapy to prevent aHUS exacerbation and received plasma therapy at least once every 2 weeks at an unchanged frequency for at least 8 weeks before first dose of OMS721.
• Exclusion Criteria:
• • Have STEC-HUS, a direct positive Coombs test, history of hematopoietic stem cell transplant, and/or HUS from an identified drug.
• • History of vitamin B12 deficiency-related HUS, systemic lupus erythematosus, and/or antiphospholipid syndrome.
• • Active cancer or history of cancer (except non-melanoma skin cancers) within 5 years of screening.
• • Have been on hemodialysis or peritoneal dialysis for greater than or equal to 12 weeks.
• • Have an active systemic bacterial or fungal infection requiring systemic antimicrobial therapy (prophylactic antimicrobial therapy administered as standard of care is allowed).
• • Baseline resting heart rate less than 45 beats per minute or greater than 115 beats per minute.
• • Baseline QTcF greater than 470 milliseconds.
• • Have malignant hypertension (diastolic blood pressure \[BP\] greater than 120 mm Hg with bilateral hemorrhages or "cotton-wool" exudates on funduscopic examination).
• • Have a poor prognosis with a life expectancy of less than three months in the opinion of the Investigator.
• • Are pregnant or lactating.
• • Have received treatment with an investigational drug or device within four weeks prior to screening.
• • Have abnormal liver function tests defined as ALT or AST \> five times ULN.
• • Have HIV infection.
• • History of cirrhosis of the liver.
• • Have previously completed treatment in an OMS721study.