Clinical Trials

Main Inclusion Criteria:

- Adult patients with evidence of thrombotic microangiopathy (TMA), including
thrombocytopenia, evidence of hemolysis, and acute kidney injury

- Vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus
influenzae infections are required prior to the start of study treatment. If the
patient has not been previously vaccinated, or if a booster is required, vaccine
should be given according to local regulations, at least 2 weeks prior to first study
drug administration. If study treatment has to start earlier than 2 weeks post
vaccination or before vaccination is given, prophylactic antibiotic treatment must be
administered at the start of study treatment and for at least 2 weeks after
vaccination

Main Exclusion Criteria:

- Treatment with complement inhibitors, including anti-C5 antibody

- ADAMTS13 deficiency (< 5% activity), and/or Shiga toxin-related hemolytic uremic
syndrome (STx-HUS), and/or Positive direct Coombs test

- Identified drug exposure-related HUS or HUS related to known genetic defects of
cobalamin C metabolism or known diacylglycerol kinase ϵ (DGKE) mediated aHUS

- Receiving PE/PI, for 28 days or longer, prior to the start of screening for the
current TMA

- Bone marrow transplantation (BMT)/hematopoietic stem cell transplantation (HSCT),
heart, lung, small bowel, pancreas, or liver transplantation

- Patients with sepsis, severe systemic infection, COVID-19 infection, systemic
infection which confounds an accurate diagnosis of aHUS or impedes the ability to
manage the aHUS disease, active infection (or history of recurrent invasive
infections) caused by encapsulated bacteria

- Kidney disease suggestive of other disease than aHUS or of chronic kidney failure or
family history of non-complement mediated genetic kidney disease

- Liver disease or liver injury at screening

- Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or
antiphospholipid antibody positivity or syndrome

- Chronic hemo- or peritoneal dialysis

Other protocol-defined inclusion/exclusion criteria may apply

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 6/8/23. Questions regarding updates should be directed to the study team contact.