Paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria
Conditions
Interventions
BCX9930
Administered orally at a dose of 200 mg twice daily for the first 2 weeks, then 400 mg twice daily
Comparator: Continued C5 inhibitor therapy
-Eculizumab
Administered by intravenous infusion
Sponsors
Sakagami Yohei
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Documented diagnosis of PNH confirmed by flow cytometry with a PNH granulocyte or monocyte clone size of >= 10% during screening. 2. Treated with a stable regimen of eculizumab for >= 3 months prior to the screening visit or ravulizumab for >= 6 months prior to the screening visit. 3. Recorded the following results during screening: a. Hemoglobin(Hb) of = 100 X 10^9 cells/L (>= 100,000 cells/microL; >= 100 G/L). c. Absolute neutrophil count of >= 0.75 X 10^9 cells/L (>= 750 cells/microL; >= 0.75 G/L). d. Platelet count of >= 30 X 10^9/L (>= 30,000/microL; >= 30 G/L). e. Adequate iron reserve based on ferritin >= Lower limit of normal(LLN) or total iron binding capacity = 60 mL/min/1.73 m^2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation and no evidence of clinically relevant abnormal renal function unrelated to underlying PNH disease.
Exclusion criteria
Exclusion criteria: 1. Known history of or existing diagnosis of hereditary complement deficiency. 2. History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation during the study. 3. Treatment with anti-thymocyte globulin within 180 days prior to the screening visit. 4. Initiation of treatment with an erythropoiesis-stimulating agent (eg, erythropoietin), a thrombopoietin receptor agonist (eg, eltrombopag), or danazol within 28 days prior to the screening visit. 5. Subjects with any of the following results at the screening visit: a. ALT (SGPT) > 3 X ULN. b. AST (SGOT) > 3 X ULN. (Note: Subjects may be enrolled with AST > 3 X ULN if explained by hemolysis.) c. Total serum bilirubin > 2 X ULN (Note: Subjects may be enrolled with total serum bilirubin > 2 X ULN if explained by hemolysis or Gilberts syndrome. In the case of hemolysis, total serum bilirubin must be < 5 X ULN and in the case of Gilberts syndrome, total serum bilirubin must be < 11 X ULN.)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in hemoglobin [mean of values at Weeks 12, 16, 20, and 24] | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Proportion of subjects who are transfusion-free [from Week 4 to Week 24] 2. Number of units of packed Red Blood Cell(pRBCs) transfused [from Week 4 to Week 24] 3. Change from baseline in FACIT-Fatigue scale score [mean of values at Weeks 12, 16, 20, and 24] | — |
Countries
Argentina, Austria, Brazil, Canada, Colombia, France, Hong Kong, Hungary, Italy, Japan, Netherlands, Slovakia, South Korea, Spain, Taiwan, Turkey, United Kingdom, United States
Contacts
Public ContactYohei Sakagami
MEDISCIENCE PLANNING INC.
Outcome results
None listed