Paroxysmal Nocturnal Hemoglobinuria PNH
Conditions
Interventions
Treatment Period 1 * The patient will receive either placebo or ALXN2040 (1:2
chance) orally three times a day for 12 weeks.
Treatment Period 2 * The patient will receive ALXN2040 orally three times
Sponsors
Alexion Pharmaceuticals
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: * Diagnosis of PNH * Clinically Evident EVH defined by: - Anemia (Hgb *9.5 gram/deciliter) with absolute reticulocyte count * 120 x 10^9/liter. - At least 1 packed red blood cell or whole blood transfusion within 6 months prior to the start of the study. * Receiving a C5 inhibitor for at least 6 months prior to Day 1 * Platelet count *30,000/microliters (*L) * Absolute neutrophil counts *750/*L. * Documentation of/or willingness to receive vaccinations and prophylactic antibiotics as required.
Exclusion criteria
Exclusion criteria: * History of a major organ transplant or hematopoietic stem cell transplantation (HSCT). * Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and/or immunosuppressants. * Known or suspected complement deficiency. * Laboratory abnormalities at screening, including: - Alanine aminotransferase >2 x ULN. - Direct bilirubin >2 x ULN (unless due to EVH or documented Gilbert's Syndrome. * Current evidence of biliary cholestasis. * Estimated glomerular filtration rate
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change From Baseline In Hemoglobin (Hgb) | — |
Secondary
| Measure | Time frame |
|---|---|
| * Percentage Of Participants With Transfusion Avoidance * Change From Baseline In Functional Assessment Of Chronic Illness Therapy (FACIT) Fatigue Scores; Scoring 0-52 * Change From Baseline In Absolute Reticulocyte Count | — |
Countries
Netherlands
Outcome results
None listed