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

A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2021210014

Enrollment

Registered

2021-06-28

Start date

2021-06-28

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria

Interventions

Additional Danicopan (ALXN2040) given to patients diagnosed with paroxysmal nocturnal hemoglobinuria and receiving complement C5 inhibitors

Double blinded

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1 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.

Exclusion criteria

Exclusion criteria: 1 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.

Design outcomes

Primary

Measure	Time frame
Change in hemoglobin (Hgb) relative to baseline after 12 weeks of treatment	—

Secondary

Measure	Time frame
Proportion of patients with transfusion avoidance	—

Countries

America, Japan

Contacts

Public ContactYuko Sugita

Alexion Pharma GK

JPDept-DevOps-PMCO@alexion.com+81-3-3457-9559

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026