atypical Hemolytic uremic syndrome (aHUS)
Conditions
Interventions
Sponsors
Maruyama Hideki
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Adult patients with evidence of thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and acute kidney injury 2. 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
Exclusion criteria
Exclusion criteria: 1. Treatment with complement inhibitors, including anti-C5 antibody 2. ADAMTS13 deficiency, and/or Shiga toxin-related hemolytic uremic syndrome (STX-HUS), and/or Positive direct Coombs test 3. Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolismor known diacylglycerol kinase epsilon (DGKE) mediated aHUS 4. Receiving PE/PI, for 28 days or longer, prior to the start of screening for the current TMA 5. Bone marrow transplantation (BMT)/hematopoietic stem cell transplantation (HSCT), heart, lung, small bowel, pancreas, or liver transplantation 6. In patients with a kidney transplant, acute kidney dysfunction consistent with the diagnosis of transplantation failure due to acute/chronic active T-Cell mediated rejection (TCMR) and/or active/chronic active antibody-mediated rejection (ABMR) according to Banff 2017 criteria 7. Liver disease or liver injury at screening 8. Patients with sepsis, severe systemic infection or COVID-19 infection 9. Presence of systemic infections (bacterial, viral, fungal or parasitic) that, in the opinion of the Investigator, confounds an accurate diagnosis of aHUS or impedes the ability to manage the aHUS disease 10. Active infection, or history of recurrent invasive infections, caused by encapsulated bacteria (i.e. meningococcus, pneumococcus), or H.influenzae 11. Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome 12. Chronic hemo- or peritoneal dialysis
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To assess the proportion of participants treated with iptacopan achieving complete thrombotic microangiopathy (TMA) response during 26 weeks of study treatment. [Complete TMA response will be assessed through (1) hematological normalization in platelet count (platelet count >= 150E9/L) and LDH (below ULN), and (2) improvement in kidney function (>=25% serum creatinine reduction from baseline), maintained for two measurements obtained at least four weeks apart and any measurement in between.] | — |
Countries
Austria, Brazil, China, Czech Republic, Greece, Guatemala, India, Japan, Korea, Russia, Slovenia, Taiwan, United Kingdom, United States
Contacts
Public ContactHideki Maruyama
Novartis Pharma. K.K.
Outcome results
None listed