Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders

This is an interventional treatment trial for Immune Thrombocytopenia (ITP) Read More

Inclusion Criteria:

All Cohorts:

• Written informed consent
• Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is 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

Additional protocol-defined inclusion / exclusion criteria may apply.