Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.

NCT05630001 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: CLNP023C123032022-502148-10-00
• Study Type: interventional
• Target: 52
• Locations: 8 countries
• Sites: 23

Brief Summary

The purpose of the study was to find out if iptacopan is effective and safe in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who switched from their current standard of care treatment (eculizumab or ravulizumab) to study treatment, iptacopan/LNP023.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

Paroxysmal Nocturnal Hemoglobinuria; iptacopan; single arm open-label; Hb≥10 g/dL in response to anti-C5 antibody; switch to iptacopan; PNH; LNP023

Outcome Measures

Primary Outcomes (1)

• Change in Hb Levels as Mean of Visits Between Day 126 and Day 168 Compared to Baseline Tested for Non-inferiority

  Change in hemoglobin (Hb) levels as mean of visits between Day 126 and Day 168 compared to baseline. Baseline is defined as as the mean of three Hb assessments conducted at the central laboratory: two during screening and the third on Day 1. The estimation of change from baseline in Hb levels was handled by the hypothetical strategy where participants were assumed as if they did not receive RBC transfusions while on treatment (RBC transfusions were expected to be rare). Assuming that participants had stable Hb levels at study entry, the mean change from baseline in Hb level between Day 126 and Day 168 was expected to be unchanged should participants have continued on anti-C5 treatment. Non-inferiority of iptacopan was therefore tested by the null hypothesis (H0) against the alternate hypothesis (H1) comparing the mean change from baseline in Hb level in iptacopan between Day 126 and Day 168 (μ) to -1 g/dL: H0: μ \<= -1, H1: μ \> -1.

  Baseline, Day 126 to Day 168

Secondary Outcomes (9)

• Change in Hb Levels as Mean of Visits Between Day 126 and Day 168 Compared to Baseline Tested for Superiority

  Baseline, Day 126 to Day 168

• Proportion of Hematological Responders to Iptacopan Treatment

  Day 126 to Day 168

• Proportion of Participants Who Remain Free From Transfusions

  Day 1 to Day 168

• Change From Baseline in Absolute Reticulocytes Count (ARC) Levels

  Baseline, Day 126 to Day 168

• Percentage Change From Baseline in Lactate Dehydrogenase (LDH) Levels

  Baseline, Day 126 to Day 168

Study Arms (1)

LNP023 200mg b.i.d.

EXPERIMENTAL

Iptacopan (LNP023) at a dose of 200 mg b.i.d. orally

Drug: Iptacopan

Interventions

Iptacopan DRUG

Treatment with iptacopan at a dose of 200 mg b.i.d. will start on the first day (Day 1) and continue for 24 weeks.

Also known as: LNP023

LNP023 200mg b.i.d.

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed informed consent must be obtained prior to participation in the study.
• Male and female participants ≥ 18 years of age, at the time of ICF signatures and with a diagnosis of PNH confirmed by treating physician.
• Stable regimen (dose and intervals) of anti-C5 antibody treatment (either eculizumab or ravulizumab) for at least 6 months prior to screening
• Mean hemoglobin level ≥10 g/dL
• Vaccination against Neisseria meningitidis and S. pneumoniae infection are required prior to the start of iptacopan treatment.
• If not received previously, vaccination against Haemophilus influenzae infections is recommended, if available and according to local regulations.
• Ability to communicate well with the investigator, to understand and comply with the requirements of the study

You may not qualify if:

• Participation in any other investigational drug trial or use of other investigational drugs at the time of enrollment
• Patients requiring red blood cell transfusion in the 6 months prior to screening or during screening
• History of stem cell transplantation or any solid organ transplantation
• Active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to study drug administration
• Presence of fever ≥ 38.0 °C (100.4 °F) within 7 days prior to study drug administration
• Human immunodeficiency virus (HIV) infection (known history of HIV or test positive for HIV antibody at Screening)
• A history of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus
• Unstable medical condition including, but not limited to, myocardial ischemia, active gastrointestinal bleeding, coexisting chronic anemia unrelated to PNH, or unstable thrombotic event not amenable to active treatment as judged by the investigator at Screening.
• History of cancer of any part of the body within the past 5 years,
• Ongoing drug or alcohol abuse that could interfere with patient's participation in the trial.
• Any medical condition deemed likely to interfere with the patient's participation in the study
• Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (23)

City Of Hope National Med Center

Duarte, California, 91010, United States

Location

USC Norris Cancer Center

Los Angeles, California, 90033, United States

Location

Lakes Research

Miami Lakes, Florida, 33014, United States

Location

Mass Gen Hosp Cancer Center

Boston, Massachusetts, 02114, United States

Location

University Of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Montefiore Medical Center

The Bronx, New York, 10461, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Prisma Health Upstate

Greenville, South Carolina, 29615, United States

Location

Huntsman Cancer Institute Univ of Utah

Salt Lake City, Utah, 84112 0550, United States

Location

Novartis Investigative Site

Nantes, 44093, France

Location

Novartis Investigative Site

Nice, 06202, France

Location

Novartis Investigative Site

Paris, 75475, France

Location

Novartis Investigative Site

Dresden, Saxony, 01307, Germany

Location

Novartis Investigative Site

Aachen, 52074, Germany

Location

Novartis Investigative Site

Essen, 45147, Germany

Location

Novartis Investigative Site

Ulm, 89081, Germany

Location

Novartis Investigative Site

Florence, FI, 50134, Italy

Location

Novartis Investigative Site

Bassano del Grappa, VI, 36061, Italy

Location

Novartis Investigative Site

Seoul, Seoul, 06351, South Korea

Location

Novartis Investigative Site

Barcelona, 08036, Spain

Location

Novartis Investigative Site

Istanbul, Fatih, 34093, Turkey (Türkiye)

Location

Novartis Investigative Site

Leeds, West Yorkshire, LS9 7TF, United Kingdom

Location

Novartis Investigative Site

London, SE5 9RS, United Kingdom

Location

Related Links

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

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

iptacopan

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@Novartis.com

+ 1 862 778 8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: A multicenter, single arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who have Hb≥10 g/dL in response to anti-C5 antibody and switch to iptacopan

Study Record Dates

• First Submitted: November 18, 2022
• First Posted: November 29, 2022
• Study Start: April 24, 2023
• Primary Completion: October 17, 2024
• Study Completion: October 17, 2024
• Last Updated: January 13, 2026
• Results First Posted: December 15, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

KR (1); ES (1); US (9); TU (1); DE (4); FR (3); GB (2); IT (2)