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

NCT ID: NCT05086744

Last Updated: 2025-10-16

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 19 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-12-21
• Study Completion Date: 2024-05-17

Brief Summary

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.

Detailed Description

This was an open-label, single-arm (within each cohort), multi-center, non-confirmatory basket study to assess the efficacy, safety and pharmacokinetics of iptacopan in participants with autoimmune benign hematological disorders. The study was set up as a basket study to allow inclusion of new cohorts (indications). The study included 2 cohorts: primary immune thrombocytopenia (ITP) and primary cold agglutinin disease (CAD). Participants in Cohort 1 (ITP) were stratified in two groups according to high/low complement activation (i.e., based on sC5b-9 level at screening). No additional cohorts were added during the study.

The study consisted of a screening period, a 12-week treatment period (Part A), a washout (for responders)/follow-up (for non-responders) period after Part A, and, for responders only, an additional treatment extension period for up to 24 months (Part B). Non-responders who had signs of clinical benefit according to the Investigator's assessment could also continue treatment with iptacopan in Part B. The washout period prior to the start of part B lasted up to 4 weeks.

Conditions

Immune Thrombocytopenia (ITP); Cold Agglutinin Disease (CAD)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Iptacopan 200 mg BID

Iptacopan 200 mg twice daily (BID) in participants with primary ITP and primary CAD

Group Type: EXPERIMENTAL

Iptacopan

Intervention Type: DRUG

Iptacopan 200 mg BID given orally (capsule)

Interventions

Iptacopan

Iptacopan 200 mg BID given orally (capsule)

Intervention Type: DRUG

Other Intervention Names

Investigational new drug; company code: LNP023

Eligibility Criteria

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

• 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

• 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.

• 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

• 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 120 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Massachusetts General Hospital

Boston, Massachusetts, United States

Novartis Investigative Site

Essen, , Germany

Novartis Investigative Site

Milan, MI, Italy

Novartis Investigative Site

Seoul, , South Korea

Novartis Investigative Site

Barcelona, Catalonia, Spain

Novartis Investigative Site

Madrid, , Spain

Novartis Investigative Site

Murcia, , Spain

Novartis Investigative Site

London, , United Kingdom

Countries

United States; Germany; Italy; South Korea; Spain; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=2615

A Plain Language Trial Summary is available on www.novctrd.com

Other Identifiers

2021-002039-40

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CLNP023L12201

Identifier Type: -

Identifier Source: org_study_id