Atypical Hemolytic Uremic Syndrome Clinical Trials

Atypical HUS results from dysregulation of the alternative complement pathway, often triggered by genetic mutations in complement pathway genes (CFH, MCP, CFI, C3, CFB, THBD) or autoantibodies against Factor H. Environmental triggers may include infections, medications, pregnancy, or malignancy. Unlike Shiga toxin-producing E.

The most common symptoms of Atypical Hemolytic Uremic Syndrome include Microangiopathic hemolytic anemia with fatigue and pallor, Thrombocytopenia causing easy bruising and petechiae, Acute kidney injury with elevated creatinine, Hematuria and proteinuria, High blood pressure, among others. Approximately 1-2 cases per million people per year; rare form of HUS accounting for 5-10% of all HUS cases. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Atypical Hemolytic Uremic Syndrome from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Research in atypical HUS has revolutionized treatment through complement pathway inhibitors that directly target the underlying cause of the disease. Trials are evaluating C5 inhibitors, Factor D inhibitors, Factor B inhibitors, and C3 inhibitors, with many examining how combination strategies might provide superior kidney protection.

As of September 2026, Trial Friend's curated drug index does not include an FDA-approved treatment specific to Atypical Hemolytic Uremic Syndrome. The Treatments section of this page checks FDA drug labels live and lists any approved drug whose label names the condition, and the trials listed below cover the investigational options.

Atypical Hemolytic Uremic Syndrome is caused by variants in any of many genes, most often CFH, CD46, CFI, C3, CFB and THBD. CFH variants are found in about 30% of cases; complement-gene variants raise risk but usually need a trigger such as infection, pregnancy or certain drugs.

It follows autosomal dominant with reduced penetrance (rarely autosomal recessive) inheritance. Approximately 1-2 cases per million people per year; rare form of HUS accounting for 5-10% of all HUS cases. A multigene panel identifies the cause in most families.

Yes. Atypical Hemolytic Uremic Syndrome can be caused by variants in many different genes (CFH, CD46, CFI, C3, CFB and THBD are among the most common), so testing is usually a multigene panel or exome sequencing rather than one gene at a time. CFH variants are found in about 30% of cases; complement-gene variants raise risk but usually need a trigger such as infection, pregnancy or certain drugs.

It follows autosomal dominant with reduced penetrance (rarely autosomal recessive) inheritance.

Knowing the exact gene matters: it decides which trials a patient qualifies for and which relatives should be tested. The labs currently offering the test are listed in the Genetic Testing section of this page, pulled live from the NIH Genetic Testing Registry. A genetic counselor can help interpret the result and decide which relatives should be tested.

Eligibility for Atypical Hemolytic Uremic Syndrome clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Atypical Hemolytic Uremic Syndrome clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.