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

NCT05086744 | Terminated Phase 2 Results FDA Drug

• 2 other identifiers: CLNP023L122012021-002039-40
• Study Type: interventional
• Target: 19
• Locations: 6 countries
• Sites: 8

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.

Conditions

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

Keywords

LNP023; Iptacopan; Immune thrombocytopenia; Cold agglutinin disease; Autoimmune benign hematological disorders

Outcome Measures

Primary Outcomes (2)

• Cohort 1 (ITP): Number of Participants With a Clinically Meaningful Response

  A study participant with ITP was considered a responder if all the below criteria were met: 1. Platelet count of ≥50 k/μL sustained for at least 2 consecutive weeks during the main, 12-week treatment part 2. Absence of rescue therapy or prohibited medications to treat ITP 3. Lack of treatment discontinuation

  Up to 12 weeks (Part A)

• Cohort 2 (CAD): Number of Participants With a Clinically Meaningful Response

  A study participant with CAD was considered a responder if all the below criteria were met: 1. Hemoglobin level increase of ≥1.5 g/dL above baseline sustained for at least 2 consecutive weeks during the main, 12-week treatment part 2. Absence of rescue therapy or prohibited medications to treat CAD 3. Lack of treatment discontinuation

  Baseline, up to 12 weeks (Part A)

Secondary Outcomes (17)

• Cohort 1 (ITP): Time to First Platelet Count ≥50 k/μL

  Up to 12 weeks (Part A)

• Cohort 2 (CAD): Time to First Hemoglobin Level ≥1.5 g/dL Above Baseline

  Baseline, up to 12 weeks (Part A)

• Cohort 1 (ITP): Duration of Time During Which Platelet Count Remains ≥50 k/μL Without the Use of Rescue Therapy

  Up to 12 weeks (Part A)

• Cohort 2 (CAD): Duration of Time During Which Hemoglobin Level Remains ≥1.5 g/dL Above Baseline Without the Use of Rescue Therapy

  Baseline, up to 12 weeks (Part A)

• Cohort 1 (ITP): Magnitude of Platelet Count Increase From Baseline

  Baseline, up to 12 weeks (Part A)

Study Arms (1)

Iptacopan 200 mg BID

EXPERIMENTAL

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

Drug: Iptacopan

Interventions

Iptacopan DRUG

Iptacopan 200 mg BID given orally (capsule)

Also known as: Investigational new drug, company code: LNP023

Iptacopan 200 mg BID

Eligibility Criteria

• Age: 18 Years - 120 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (8)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Novartis Investigative Site

Essen, 45147, Germany

Location

Novartis Investigative Site

Milan, MI, 20122, Italy

Location

Novartis Investigative Site

Seoul, 06351, South Korea

Location

Novartis Investigative Site

Barcelona, Catalonia, 08035, Spain

Location

Novartis Investigative Site

Madrid, 28009, Spain

Location

Novartis Investigative Site

Murcia, 30008, Spain

Location

Novartis Investigative Site

London, W12 0HS, United Kingdom

Location

Related Publications (1)

• Roth A, Barcellini W, Ademokun C, Jang J, Lozano ML, Ferreiras DV, Pascual-Izquierdo C, Chitnis S, Matviykiv S, Vitaliti A, Chen C, Katsanou V, Chawla R, Al-Samkari H. Iptacopan for Immune Thrombocytopenia and Cold Agglutinin Disease: A Global Phase 2 Basket Clinical Trial. Am J Hematol. 2026 Feb;101(2):242-254. doi: 10.1002/ajh.70147. Epub 2025 Dec 9.

  PMID: 41366634 DERIVED

Related Links

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

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Anemia, Hemolytic, Autoimmune

Interventions

iptacopan; Drugs, Investigational

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Anemia, Hemolytic; Anemia

Intervention Hierarchy (Ancestors)

Pharmaceutical Preparations

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@novartis.com

862-778-8300

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a basket study with different Cohorts.

Study Record Dates

• First Submitted: September 28, 2021
• First Posted: October 21, 2021
• Study Start: December 21, 2021
• Primary Completion: September 20, 2023
• Study Completion: May 17, 2024
• Last Updated: October 16, 2025
• Results First Posted: March 19, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share

Locations

DE (1); IT (1); US (1); GB (1); KR (1); ES (3)