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JRCT ID: jRCT2021210014

Registered date:28/06/2021

Pivotal study of danicopan as add-on therapy to a C5 inhibitor (eculizumab or ravulizumab) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have Clinically Evident EVH

Basic Information

Recruitment status Recruiting
Health condition(s) or Problem(s) studiedParoxysmal Nocturnal Hemoglobinuria
Date of first enrollment28/06/2021
Target sample size84
Countries of recruitmentAmerica,Japan
Study typeInterventional
Intervention(s)Additional Danicopan (ALXN2040) given to patients diagnosed with paroxysmal nocturnal hemoglobinuria and receiving complement C5 inhibitors


Primary OutcomeChange in hemoglobin (Hgb) relative to baseline after 12 weeks of treatment
Secondary OutcomeProportion of patients with transfusion avoidance

Key inclusion & exclusion criteria

Age minimum>= 18age old
Age maximumNot applicable
Include criteria1 Diagnosis of PNH 2 Clinically Evident Extravascular hemolysis defined by: Anemia, Hgb is 9.5 g per dL or less than 9.5, with absolute reticulocyte count 120000 per microL or more. At least 1 packed RBC or whole blood transfusion within 6 months prior to the start of the study 3 Receiving a C5 inhibitor for at least 6 months prior to Day 1 in this study at an approved dose or higher and with no change in dose or interval for at least 24 weeks preceding Day 1. For those patients who recently switched from eculizumab to ravulizumab, they must have received at least the loading dose and 3 maintenance doses of ravulizumab preceding Day 1. 4 Platelet count 30,000 per microL or more than 30,000 per microL without the need for platelet transfusions 5 Absolute neutrophil counts 750 per microL or more 6 Documentation of vaccination for Neisseria meningitidis: All patients must be vaccinated against meningococcal infections within 3 years prior to, or at the time of initiating study Age 18 years or older.
Exclude criteria1 History of a major organ transplant or hematopoietic stem cell transplantation 2 Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and or immunosuppressants 4 Known or suspected complement deficiency 8 Laboratory abnormalities at screening, including: Alanine aminotransferase more than 2 x ULN Direct bilirubin more than 2 x ULN 11 Current evidence of biliary cholestasis 13 Estimated glomerular filtration rate less than 30 mL per min per 1.73 m2 and or are on dialysis.

Related Information


Public contact
Name Takeo Kamata
Address 1-18-14 Ebisu Shibuya-ku Tokyo Japan 150-0013
Telephone +81-3-57950-0775
Affiliation Alexion Pharma GK
Scientific contact
Name Takeo Kamata
Address 1-18-14 Ebisu Shibuya-ku Tokyo Japan 150-0013
Telephone +81-3-5795-0775
Affiliation Alexion Pharma GK