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

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

ClinicalTrials.gov Identifier: NCT05086744

Novartis Reference Number: CLNP023L12201

Last Update: Apr 22, 2022

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

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)
Phase 
Phase 2
Overall status 
Recruiting
Start date 
Dec 21, 2021
Completion date 
Nov 03, 2025
Gender 
All
Age(s)
18 Years and older (Adult, Older Adult)

Interventions

Drug
Iptacopan
Iptacopan 200 mg BID

Eligibility Criteria

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 prior line of ITP-directed therapy
Sustained thrombocytopenia

Cohort 2 specific inclusion criteria:

Participants with a diagnosis of primary CAD
Participants must have received at least 1 prior line of CAD-directed therapy
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 a thrombopoietin receptor agonist or low-dose corticosteroid, as long as stable dosage for at least 4 weeks prior to baseline
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.

Study Locations

Germany
Novartis Investigative Site
Recruiting
Essen, 45147
-
Germany
Korea, Republic of
Novartis Investigative Site
Recruiting
Seoul, 06351
-
Korea, Republic of
Spain
Novartis Investigative Site
Recruiting
Barcelona, 08035
Catalunya
Spain
Novartis Investigative Site
Recruiting
Madrid, 28009
-
Spain
Novartis Investigative Site
Recruiting
Murcia, 30008
-
Spain
United Kingdom
Novartis Investigative Site
Recruiting
London, W12 0HS
-
United Kingdom

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
Name: 
Novartis Pharmaceuticals
Phone: 

Have a question?

Call 1-888-669-6682 or email [email protected]