Paroxysmal nocturnal hemoglobinuria PNH
Conditions
Interventions
Arm A : LNP023 monotherapy
Arm B : Anti-C5 antibody treatment(Eculizumab or Ravulizumab)
Sponsors
Suzuki Kazuyuki
Eligibility
Sex/Gender
All
Inclusion criteria
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
Exclusion criteria: - Participants on a stable eculizumab dose but with a dosing interval of 11 days or less - 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 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., 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - Percentage of participants achieving a sustained increase in hemoglobin levels of >= 2 g/dL in the absence of red blood cell transfusions - Percentage of participants achieving sustained hemoglobin levels >= 12 g/dL in the absence of red blood cell transfusions | — |
Countries
Japan, Netherlands
Contacts
Public ContactKazuyuki Suzuki
Novartis Pharma. K.K.
Outcome results
None listed