Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

Completed · Phase 3

Conditions studied: Paroxysmal Nocturnal Hemoglobinuria (PNH)

In brief

This study was a multi-center, randomized, open-label, active comparator-controlled, parallel group study. The purpose of this Phase 3 study in PNH patients presenting with residual anemia despite treatment with anti-C5 antibody, was to determine whether iptacopan is efficacious and safe for the treatment of PNH through demonstration of superiority of iptacopan compared to anti-C5 antibody treatment.

Key facts

Study ID

NCT04558918

Run by

Novartis Pharmaceuticals

People needed

97

Starts

2021-01-25

Expected to finish

2023-03-06

Last updated by the study team

2024-10-09

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male and female participants ≥ 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size ≥ 10%
• Stable regimen of anti-C5 antibody treatment (either eculizumab or ravulizumab) for at least 6 months prior to randomization
• Mean hemoglobin level <10 g/dL
• Vaccination against Neisseria meningitidis infection is required prior to the start of treatment.
• If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given

You may not qualify if…

• Participants on a stable eculizumab dose but with a dosing interval of 11 days or less or patients on stable ravulizumab dose but with a dosing interval of less than 8 weeks.
• Known or suspected hereditary complement deficiency at screening
• History of hematopoietic stem cell transplantation
• Patients with laboratory evidence of bone marrow failure (reticulocytes <100x10E9/L; platelets <30x10E9/L; neutrophils <500x10E6/L).
• Active systemic bacterial, viral (incl. COVID-19), or fungal infection within 14 days prior to study drug administration
• A history of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
• Major concurrent comorbidities including but not limited to severe kidney disease (e.g., eGFR < 30 mL/min/1.73 m2, dialysis), advanced cardiac disease (e.g., NYHA class IV), severe pulmonary disease (e.g., severe pulmonary hypertension (WHO class IV)), or hepatic disease (e.g., active hepatitis) that in the opinion of the investigator precludes participant's participation in the study.

Where it is running

• City Of Hope National Med Center City of Hope Medical Center — Duarte, California, United States
• Univ Cali Irvine ALS Neuromuscular — Orange, California, United States
• Augusta University Georgia Patient Treatment — Augusta, Georgia, United States
• Levine Cancer Insitute Carolinas Healthcare System — Charlotte, North Carolina, United States
• Cleveland Clinic Foundation Dept.ofTaussigCancer Center — Cleveland, Ohio, United States
• Novartis Investigative Site — Santo André, São Paulo, Brazil
• Novartis Investigative Site — São Paulo, São Paulo, Brazil
• Novartis Investigative Site — Ostrava, Poruba, Czechia
• Novartis Investigative Site — Lille, France
• Novartis Investigative Site — Paris, France
• Novartis Investigative Site — Toulouse, France
• Novartis Investigative Site — Aachen, Germany
• Novartis Investigative Site — Essen, Germany
• Novartis Investigative Site — Hamburg, Germany
• Novartis Investigative Site — Riesa, Germany
• Novartis Investigative Site — Ulm, Germany
• Novartis Investigative Site — Ascoli Piceno, AP, Italy
• Novartis Investigative Site — Avellino, AV, Italy
• Novartis Investigative Site — Florence, FI, Italy
• Novartis Investigative Site — Milan, MI, Italy
• Novartis Investigative Site — Roma, RM, Italy
• Novartis Investigative Site — Torino, TO, Italy
• Novartis Investigative Site — Bassano del Grappa, VI, Italy
• Novartis Investigative Site — Nagoya, Aichi-ken, Japan
• Novartis Investigative Site — Fukushima, Fukushima, Japan

Full record on ClinicalTrials.gov

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