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

  • End date
    Nov 3, 2025
  • participants needed
  • sponsor
    Novartis Pharmaceuticals
Updated on 7 October 2022


The main purpose of this study is 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.

Condition Immune Thrombocytopenia (ITP), Cold Agglutinin Disease (CAD)
Treatment Iptacopan
Clinical Study IdentifierNCT05086744
SponsorNovartis Pharmaceuticals
Last Modified on7 October 2022


Yes No Not Sure

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
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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