Myelodysplastic Syndrome Clinical Trial
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab
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.
The purpose of this study is to determine the effectiveness of danicopan in improving anemia, as measured by increased blood hemoglobin, when given with eculizumab (a drug commonly used for treatment of PNH) for 24 weeks in participants with PNH.
The 24-week treatment period was followed by a long-term extension phase. In the extension phase, participants received the same danicopan dose plus eculizumab as they were receiving at the end of 24-week treatment phase.
Results are reported for the 24-week treatment period.
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.
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There are 2 Locations for this study
Baltimore Maryland, 21287, United States
Cleveland Ohio, 44195, United States
Florence , , Italy
Naples , , Italy
London , , United Kingdom
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