Evaluating OMS721 for treating atypical hemolytic uremic syndrome

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

This study is testing a new treatment called OMS721 to see if it can help adults and teens with atypical hemolytic uremic syndrome feel better and improve their health.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 80 (estimated)
• Ages: 12 Years and up
• Sex: All
• Sponsor: Omeros Corporation (Industry-sponsored)
• Locations: 2 sites (Los Angeles, California and 1 other locations)
• Trial ID: NCT03205995 on ClinicalTrials.gov

What this trial studies

This Phase 3, multicenter study aims to assess the safety and efficacy of OMS721 in adults and adolescents diagnosed with atypical hemolytic uremic syndrome (aHUS). The study will measure changes in platelet counts and evaluate pharmacokinetics, pharmacodynamics, and anti-drug antibody responses. Participants will undergo a structured treatment regimen consisting of screening, treatment induction, maintenance, and follow-up periods. Approximately 80 subjects will be enrolled, with an interim analysis planned after 40 participants complete 26 weeks of treatment.

Who should consider this trial

Good fit: Ideal candidates include adults and adolescents aged 12 and older with a clinical diagnosis of primary atypical hemolytic uremic syndrome who are either plasma therapy-resistant or plasma therapy-responsive.

Not a fit: Patients with STEC-HUS, a direct positive Coombs test, or those with a history of hematopoietic stem cell transplant may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve outcomes for patients with atypical hemolytic uremic syndrome.

How similar studies have performed: Other studies have shown promise in treating atypical hemolytic uremic syndrome with novel therapies, but this specific approach with OMS721 is relatively novel.

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.

Where this trial is running

Los Angeles, California and 1 other locations

• Omeros Investigational Site — Los Angeles, California, United States (Not_yet_recruiting)
• Omeros Investigational Site — Chicago, Illinois, United States (Recruiting)

Study contacts

• Study coordinator: Alan Lew

Conditions Thrombotic Microangiopathies; Atypical Hemolytic Uremic Syndrome; TMA; aHUS