BCX9930 for the Treatment of PNH in Subjects Not Receiving Other Complement Inhibitor Therapy (REDEEM-2)

JRCT ID: jRCT2051210180

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: 26/11/2021
• Target sample size: 57
• Countries of recruitment: Albania,Japan,Argentina,Japan,Azerbaijan,Japan,Brazil,Japan,Canada,Japan,China,Japan,Colombia,Japan,Czech Republic,Japan,Hong Kong,Japan,Italy,Japan,Lithuania,Japan,Malaysia,Japan,Philippines,Japan,Poland,Japan,Romania,Japan,Serbia,Japan,South Africa,Japan,South Korea,Japan,Spain,Japan,Sweden,Japan,Taiwan,Japan,Turkey,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

BCX9930 will be administered orally at 200 mg BID, approximately 12 hours apart, using tablets for the first 2 weeks, then 400 mg BID, approximately 12 hours apart.

Placebo
Administered orally twice daily

Outcome(s)

• Primary Outcome: Change from baseline in Hemoglobin [at Week 12]

Secondary Outcome

1. Proportion of subjects who are transfusion-free [from Week 4 to Week 12]
2. Number of units of packed Red Blood Cell(pRBCs) transfused [from Week 4 to Week 12]
3. Percent change from baseline in Lactate dehydrogenase(LDH) [at Week 12]
4. Change from baseline in FACIT-Fatigue scale score [at Week 12]

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. Are either:
(a) naive to treatment with a complement inhibitor, or
(b) have received no treatment with a complement inhibitor for at least 12 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.
4. Do not have access to or have a contraindication (ie, have had a serious adverse reaction) to approved complement, C5 or C3, inhibitor therapies.

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): NCT05116787