BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy (REDEEM-1)

JRCT ID: jRCT2051210179

Registered date:23/02/2022

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Paroxysmal nocturnal hemoglobinuria
• Date of first enrollment: 07/01/2022
• Target sample size: 81
• Countries of recruitment: Argentina,Japan,Austria,Japan,Brazil,Japan,Canada,Japan,Colombia,Japan,France,Japan,Hong Kong,Japan,Hungary,Japan,Italy,Japan,Netherlands,Japan,Slovakia,Japan,South Korea,Japan,Spain,Japan,Taiwan,Japan,Turkey,Japan,United Kingdom,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

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 per current dose regimen
-Ravulizumab
Administered as intravenous infusion per current dose regimen

Outcome(s)

• Primary Outcome: Change from baseline in hemoglobin [mean of values at Weeks 12, 16, 20, and 24]

Secondary Outcome

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]

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include 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 <= 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.

Exclude 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.)

Related Information

• Primary Sponsor: Sakagami Yohei
• Secondary ID(s): NCT05116774