An Open-label, Multi-center, Phase 2 Basket Study to Assess Efficacy, Safety and Pharmacokinetics of Iptacopan (LNP023) in Participants With Autoimmune Benign Hematological Disorders
The main purpose of this study was to evaluate the efficacy and safety of iptacopan in participants with autoimmune benign hematological disorders such as primary immune thrombocytopenia and primary cold agglutinin disease.
⁃ All Cohorts:
• Written informed consent
• Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections was required and vaccination against Haemophilus influenzae infection is recommended prior to the start of treatment.
• Weight of at least 35 kg
⁃ Cohort 1 specific inclusion criteria:
• Participants with a diagnosis of persistent or chronic primary ITP
• Participants must have received at least 1 unique prior therapy administered with the intention to treat ITP
• Sustained thrombocytopenia
⁃ Cohort 2 specific inclusion criteria:
• Participants with a diagnosis of primary CAD
• Participants must have received at least 1 unique prior therapy administered with the intention to treat CAD
• Laboratory evidence of ongoing hemolysis
• Sustained anemia