Proof of Concept Study of ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH)
Status:
Recruiting
Trial end date:
2024-06-30
Target enrollment:
Participant gender:
Summary
The study will evaluate the efficacy and safety of the oral Factor D (FD) inhibitor ALXN2050
(ACH-0145228) monotherapy in patients with PNH that are treatment naïve, or patients
currently treated with eculizumab who still experience anemia and reticulocytosis, or
patients currently treated with ALXN2040 (danicopan) as monotherapy. After signing consent,
participants will have periodic visits through Week 12, at which time the primary endpoint
and key secondary assessments will be analyzed. Participants will continue on treatment past
12 weeks into a long-term extension portion of the trial.