Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy
Paroxysmal Nocturnal Hemoglobinuria (PNH)
About this trial
This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria (PNH) focused on measuring paroxysmal nocturnal hemoglobinuria, PNH, extravascular hemolysis, EVH, factor D inhibitor, complement, C5 inhibitor, danicopan
Eligibility Criteria
Key Inclusion Criteria:
- Diagnosis of PNH.
- Male or female, ≥ 18 years of age
Eligibility Criteria:
Eligibility Criteria Specific for Group 1:
- PNH Patients who have no history of treatment with any complement inhibitor at any dose.
- PNH Type III erythrocyte or granulocyte clone size ≥10%
- Absolute reticulocyte count ≥100×10^9/liter [L].
- Anemia (Hgb <10.5 grams/deciliter [g/dL]).
- LDH ≥1.5× upper limit of normal.
- Platelet count ≥30,000/microliter (µL)
- Absolute neutrophil count (ANC) ≥750/ µL.
Eligibility Criteria Specific for Group 2:
- Stable background regimen of at least 24 weeks for eculizumab without change in dose or interval for at least the past 8 weeks
- Anemia (Hgb <10 g/dL)
- Absolute reticulocyte count ≥100×10^9/L
- Platelet count ≥30,000/µL
- Absolute neurophil count (ANC) ≥750/ µL
Eligibility Criteria Specific for Group 3:
1. Patient received danicopan during Study ACH471-103
Key Exclusion Criteria:
- History of a major organ transplant or hematopoietic stem cell/marrow transplant .
- Known aplastic anemia or other bone marrow failure that requires HSCT, or if these patients are on immunosuppressive agents for less than 24 weeks.
- Known underlying bleeding disorders or any other conditions leading to anemia not primarily associated with PNH.
- Estimated glomerular filtration rate <30 milliliters/minute/1.73 meters squared and/or are on dialysis.
Sites / Locations
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
Arms of the Study
Arm 1
Experimental
Open-label ALXN2050 Monotherapy
Experimental: Open-label ALXN2050 Monotherapy ALXN2050 orally administered Group 1: Patients with PNH who are treatment naïve Group 2: Patient with PNH who have received complement component 5 (C5) inhibition with eculizumab for at least 6 months, who continue to experience anemia and reticulocytes above the upper limit of normal (ULN) Group 3: Patients with PNH who have received danicopan monotherapy during study ACH471-103