Experimental drug iptacopan tested for rare blood disorders

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 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 Exclusion Criteria: All cohorts: * Use of other investigational drugs at the time of enrollment, or within 5 half-lives of enrollment, or within 30 days, whichever is longer; or longer if required by local regulations * Past or concomitant use of medications prohibited by the protocol * Known or suspected hereditary or acquired complement deficiency * History of primary or secondary immunodeficiency, including a positive HIV test result * Chronic infection with Hepatitis B or C virus * History of recurrent invasive infections caused by encapsulated organisms, including Neisseria meningitidis, Streptococcus pneumoniae, or Haemophilus influenzae * Presence or suspicion of any active infection within 14 days prior to first study drug administration. * Any medical condition deemed likely to interfere with the participant's participation in the study * Any malignant disease diagnosed within the past 5 years, with the exception of localized non-melanoma skin cancer, in situ cervical cancer, or, for CAD, a low-grade lymphoproliferative disorder. * History of bone marrow/hematopoietic stem cell or solid organ transplantation. * Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after last iptacopan dose * Active severe bleeding or history of intracranial hemorrhage. * Liver disease, or liver injury as indicated by abnormal liver function tests. * Severe concurrent comorbidities of unstable medical conditions. Cohort 1 specific exclusion criteria: * Secondary ITP, as may arise in the setting of certain autoimmune disorders, immunodeficiency syndromes, infections, malignancies, and drug treatments * No ITP-directed background therapy permitted, with the exception of either a thrombopoietin receptor agonist or low-dose corticosteroid, as long as stable dosage for at least 4 weeks prior to first iptacopan dose * Abnormal coagulation screening labs Cohort 2 specific exclusion criteria: * Secondary cold agglutinin syndrome, as may arise in the setting of certain infections, autoimmune disorders, and malignancies (with the exception of a low-grade lymphoproliferative disorder) * No CAD-directed background therapy permitted