A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult Patients With PNH and Residual Anemia, Despite Treatment With an Intravenous Anti-C5 Antibody.

CLNP023C12302

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.

2021-01-25

2022-09-26

2023-03-06

Completed

Early phase I

97

Inclusion Criteria: * 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 Exclusion Criteria: * 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.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 97, 'type': 'ACTUAL'}}

2024-04-02