A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy
Latest Information Update: 25 Nov 2024
At a glance
- Drugs Vemircopan (Primary)
- Indications Paroxysmal nocturnal haemoglobinuria
- Focus Proof of concept; Therapeutic Use
- Sponsors Achillion Pharmaceuticals; Alexion AstraZeneca Rare Disease
Most Recent Events
- 06 Jun 2024 Status changed from active, no longer recruiting to discontinued, because the study drug did not appropriately control IVH; significantly increased rates of BTH and LDH excursions (LDH values > X2ULN).
- 11 Apr 2024 This trial has been discontinued in Italy, according to European Clinical Trials Database record.
- 21 Mar 2024 This trial has been discontinued in Spain, according to an European Clinical Trials Database.