Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab

• Status: Active, not recruiting
• Trial end date: 2022-02-28
• Target enrollment: 0
• Participant gender: All

Summary

To determine the effectiveness of ACH-0144471 (also known as danicopan and ALXN2040) in improving anemia when given with eculizumab for 24 weeks in participants with PNH. Danicopan dose may be increased within each participant, to a maximum of 200 milligrams (mg) three times daily (TID) based on safety and efficacy at protocol-specified time points.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Achillion Pharmaceuticals; Alexion Pharmaceuticals
• Collaborator: Achillion, a wholly owned subsidiary of Alexion
• Treatments: Eculizumab

Criteria

Key Inclusion Criteria:

• Diagnosed with PNH

• Have received at least one red blood cell transfusion within last 12 weeks

• Anemia with adequate reticulocytosis

• Must be on a stable regimen of eculizumab

• Platelet count ≥ 40,000/microliter without the need for platelet transfusions

• Documentation of vaccination for Neisseria meningitidis, Haemophilus influenza, and Streptococcus pneumoniae or willingness to receive vaccinations based on local guidelines

• Willingness to receive antibiotic prophylaxis

• Female participants must use highly effective birth control to prevent pregnancy during the clinical trial and for 30 days after their last dose of study drug

• Male participants must use a highly effective birth control with a female partner to prevent pregnancy during the clinical trial and for 90 days after the last dose of study drug

Key Exclusion Criteria:

• Current evidence of bone marrow failure or aplastic anemia requiring treatment

• History of a major organ transplant or hematopoietic stem cell/marrow transplant

• Received another investigational agent within 30 days or 5 half-lives of the investigational agent prior to study entry, whichever is greater

• Documented C5 complement protein mutations

• Known or suspected complement deficiency

• Contraindication to any of the required vaccinations

• Active bacterial infection or clinically significant active viral infection, a body temperature >38°C, or other evidence of infection

• History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection

• History of hypersensitivity reactions to commonly used antibacterial agents

Note: Additional inclusion/exclusion criteria may apply, per protocol.