hemolysis red blood cells PNH
Conditions
Interventions
LNP023 200mg bid
Sponsors
Novartis
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: - Male and female participants >= 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with RBCs and WBCs granulocyte/monocyte 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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Endpoint(s) for primary objective(s) • Response defined as having an increase from baseline in Hb >= 2 g/dL assessed between Day 126 and Day 168, in the absence of packed red blood cell transfusions between Day 14 and Day 168 • Response defined as having Hb >= 12 g/dL between Day 126 and Day 168 in the absence of packed-red blood cell transfusions between Day 14 and Day 168 | — |
Secondary
| Measure | Time frame |
|---|---|
| Endpoint(s) for secondary objective(s) • Absence of administration of packed-red blood cell transfusions between Day 14 and Day 168 • Change from baseline in hemoglobin (g/dL) as mean of visits between Day 126 and Day 168 • Change from baseline in FACIT-Fatigue scores as mean of visits between Day 126 and Day 168 • Change from baseline in reticulocyte count (109/L) as mean of visits between Day 126 and Day 168 • Percent change from baseline in LDH levels (U/L) as mean of visits between Day 126 and Day 168 • Occurrences of breakthrough hemolysis reported between Day 1 and Day 168 • Occurrences of MAVEs occurring between Day 1 and Day 168 | — |
Countries
Netherlands
Outcome results
None listed