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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

Trial Profile

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

Status: Discontinued
Phase of Trial: Phase II

Latest Information Update: 25 Nov 2024

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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.

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