An Open Label, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of APL-2 as an Add-On to Standard of Care in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Trial Profile

An Open Label, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of APL-2 as an Add-On to Standard of Care in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Active, no longer recruiting
Phase of Trial: Phase I

Latest Information Update: 04 Jul 2018

At a glance

  • Drugs APL 2 (Primary)
  • Indications Paroxysmal nocturnal haemoglobinuria
  • Focus Adverse reactions; Proof of concept
  • Acronyms PHAROAH
  • Sponsors Apellis Pharmaceuticals
  • Most Recent Events

    • 26 Jun 2018 Results presented in the Apellis Pharmaceuticals media release.
    • 26 Jun 2018 According to an Apellis Pharmaceuticals media release, 32 week data from this trial was released in December 2017 at the International PNH Interest Group meeting, with six patients enrolled to receive co-treatment of eculizumab and APL-2 at 270 mg/day.
    • 08 Dec 2017 Results presented in an Apellis Pharmaceuticals Media Release.
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