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

COMPLETEDPHASE3

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

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

NCT05630001•Novartis Pharmaceuticals

View on ClinicalTrials.gov

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.

Detailed Description

This was a multicenter, single-arm, open label trial, with iptacopan treatment for 24 weeks in adult PNH patients. This study was comprised of two periods: * A Screening period lasting up to 8 weeks. * A 24-week open-label, iptacopan Treatment period. After completion of the treatment period, participants who continued to benefit from the iptacopan treatment based on the study doctor's evaluation were able to join the Roll-over extension study (CLNP023C12001B).

Eligibility

Age

18 - 100 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•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

Exclusion Criteria

•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.
+2 more criteria

Locations (23)

City Of Hope National Med Center

Duarte, California, United States

USC Norris Cancer Center

Los Angeles, California, United States

Lakes Research

Miami Lakes, Florida, United States

Mass Gen Hosp Cancer Center

Boston, Massachusetts, United States

University Of Minnesota

Minneapolis, Minnesota, United States

Montefiore Medical Center

The Bronx, New York, United States

Cleveland Clinic Foundation

Cleveland, Ohio, United States

Prisma Health Upstate

Greenville, South Carolina, United States

Huntsman Cancer Institute Univ of Utah

Salt Lake City, Utah, United States

Novartis Investigative Site

Nantes, France

Key Dates

Start Date

April 24, 2023

Primary Completion Date

October 17, 2024

Completion Date

October 17, 2024

Last Updated

January 13, 2026

Enrollment

ACTUAL participants

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Interventions

Iptacopan

DRUG

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

NCT03520647

Severe Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)+1 more

View study

RECRUITING

National Longitudinal Cohort of Hematological Diseases

NCT04645199

Multiple MyelomaAcute Myeloid Leukemia+15 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 13, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

National Longitudinal Cohort of Hematological Diseases

Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment

Home Reported Outcomes in PNH

Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria

A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH