A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy(REEDEM-1)

Sakagami Yohei0 个研究点目标入组 81 人开始时间: 2022年2月23日最近更新: 2023年10月17日

研究概览

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•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 <= 105 g/L (<= 10.5 g/dL).
•b. Absolute Reticulocyte Count(ARC) 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 <= Upper limit of normal (ULN).
•f. Estimated glomerular filtration rate(eGFR) of >= 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.

•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.)

发起方

Sakagami Yohei