Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy

Launched by NOVARTIS PHARMACEUTICALS · May 13, 2021

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called iptacopan (also known as LNP023) for adults with a condition called atypical hemolytic uremic syndrome (aHUS). This study aims to find out if iptacopan is effective and safe for patients who have never received treatment with complement inhibitors before. This is a critical phase of research that could help improve how this serious condition is managed.

To be eligible for the trial, participants must be adults showing signs of active thrombotic microangiopathy (TMA), which includes low platelet counts, hemolysis (breaking down of red blood cells), and kidney problems. Participants will need to receive certain vaccinations before starting the trial to protect against specific infections. Those with a history of certain other conditions, previous treatments with complement inhibitors, or other specific health issues will not be eligible. If you join the trial, you will be monitored closely and receive the trial treatment, with the hope that it can help manage your condition more effectively.

Gender

ALL

Eligibility criteria

• Main Inclusion Criteria:
• • Adult patients with evidence of active 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 (\<10% activity or \<0.1U/ml), 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 14 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 or active severe systemic bacterial, viral (including COVID-19) or fungal 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