Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1 Level: PT Classification code 10034042 Term: Paroxysmal nocturnal haemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Sponsors
Novartis Pharma AG
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 =65 years) yes F.1.3.1 Number of subjects for this age range 9
Exclusion criteria
Exclusion criteria: - Participants on a stable eculizumab dose but with a dosing interval of 11 days or less or participants 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 <100x109/L; platelets <30x109/L; neutrophils <500x106/L). - Active systemic bacterial, viral (including 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 m^2, 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. Other protocol-defined exclusion criteria may apply
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - Proportion of participants achieving a sustained increase in hemoglobin levels of = 2 g/dL in the absence of red blood cell transfusions - Proportion of participants achieving sustained hemoglobin levels = 12 g/dL in the absence of red blood cell transfusions.;Primary end point(s): - Proportion of participants achieving a sustained increase in hemoglobin levels from baseline of = 2 g/dL in the absence of red blood cell transfusions - Proportion of participants achieving sustained hemoglobin levels = 12 g/dL in the absence of red blood cell transfusions.;Timepoint(s) of evaluation of this end point: Day 126 and Day 168;Secondary Objective: - Proportion of participants who remain free from transfusions - Average change in hemoglobin - Change in fatigue score, using the FACIT-Fatigue questionnaire - Average change in reticulocyte counts - Average percent change in LDH - Rate of breakthrough hemolysis (BTH) - Rates of Major Adverse Vascular Events (MAVEs incl. thrombosis | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Proportion of participants who remain free from transfusions 2. Change from baseline in hemoglobin (g/dL) as mean of visits between Day 126 and D168 3. Change from baseline in FACIT-Fatigue scores as mean of visits between Day 126 and Day 168 4. Change from baseline in reticulocyte counts as mean of visits between Day 126 and Day 168 5. Percent change from baseline in LDH levels (U/L) as mean of visits between Day 126 and Day 168 6. Rate of breakthrough hemolysis (BTH) 7. Rates of Major Adverse Vascular Events (MAVEs incl. thrombosis;Timepoint(s) of evaluation of this end point: 1. Day 14 and Day 168 2. Baseline and as mean of visit Day 126, 140, 154 and 168. 3. Baseline and as mean of visit Day 126, 140, 154 and 168. 4. Baseline and as mean of visit Day 126, 140, 154 and 168. 5. Baseline and as mean of visit Day 126, 140, 154 and 168. 6. Day 1 and Day 168. 7. Day 1 and Day 168. | — |
Countries
Brazil, Canada, Czechia, Czech Republic, France, Germany, Hungary, Italy, Japan, Korea, Republic of, Netherlands, Spain, Taiwan, United Kingdom, United States
Contacts
Public ContactMedizinischer Infoservice (MCC)
Novartis Pharma GmbH
Outcome results
None listed