Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab (PNH)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
About this trial
This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria (PNH) focused on measuring PNH, Paroxysmal Nocturnal Hemoglobinuria, ACH-0144471, Danicopan, Eculizumab, ALXN2040
Eligibility 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.
Sites / Locations
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Experimental
Experimental
Experimental
Experimental
Group 1: 100 mg Danicopan TID + Eculizumab
Group 2: Initial dose 100 or 150 mg Danicopan TID + Eculizumab
Group 3: Initial dose of 100, 150, or 200 mg Danicopan TID + Eculizumab
Group 4: Optimal Dose of Danicopan TID + Eculizumab
Starting dose of 100 mg danicopan TID in combination with eculizumab.
Starting dose of 100 or 150 mg danicopan TID in combination with eculizumab.
Starting dose of 100, 150, or 200 mg danicopan TID in combination with eculizumab.
Optimal dose (starting dose of either 100, 150, or 200 mg, as determined from Groups 1-3) of danicopan TID in combination with eculizumab.