PEGASUS - APL2-302/Version 3.0/30Mar2018

  • Research type

    Research Study

  • Full title

    A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

  • IRAS ID

    249506

  • Contact name

    Pascal Deschatelets

  • Contact email

    pascal@apellis.com

  • Sponsor organisation

    Apellis Pharmaceuticals, Inc.

  • Eudract number

    2017-004268-36

  • Clinicaltrials.gov Identifier

    NCT03500549

  • Clinicaltrials.gov Identifier

    123087, US IND Number

  • Duration of Study in the UK

    2 years, 6 months, 1 days

  • Research summary

    The “Pegasus”-study APL2-302 is a multinational phase-III-study sponsored by Apellis Pharmaceuticals Inc. It is a prospective, randomized, multi-center, open-label, active-comparator (active drug) controlled study. The purpose of this research study is to evaluate the efficacy and safety of the new investigational drug APL-2 for subcutaneous infusion in comparison to intravenous eculizumab in treatment of patients with paroxysmal nocturnal haemoglobinuria (PNH), who still have anaemia despite continuous treatment with eculizumab, the current standard of care treatment. PNH is a rare disorder that causes the red blood cells to break down too early. Many PNH patients suffer from complications such as blood clots or low red blood cell counts (anaemia) even after receiving a standard treatment.
    As shown by previous clinical studies, APL-2 provides sustained inhibition of haemolytic (destruction of red blood cells) activity in PNH patients who have never received eculizumab, and in patients receiving eculizumab who continue to be anaemic (Haemoglobin (Hb) <10.5 g/dL). To date, no safety signals have emerged from ongoing studies in PNH patients that preclude further development. Thus, the aim of the proposed study is to confirm efficacy of subcutaneous APL-2 as monotherapy for the treatment of PNH (Hb-level after 16 weeks).
    Study population includes approximately 70 adult, male and female subjects diagnosed with PNH. After a 4-week run-in period, where patients receive APL-2 plus eculizumab, in a following 16-weeks controlled period participants will be randomly divided into two treatment groups: 35 patients into the APL-2 group and 35 patients receiving eculizumab therapy. After this all subjects will continue into a 32-week open-label period with twice-weekly doses of APL-2. Patients gaining benefit from APL-2-treatment will then be offered entry into an open label extension study, or otherwise complete a 6-week follow-up phase. The complete study duration per patient is at maximum 72 weeks (or every 3 days).

  • REC name

    Yorkshire & The Humber - Leeds East Research Ethics Committee

  • REC reference

    18/YH/0311

  • Date of REC Opinion

    10 Dec 2018

  • REC opinion

    Further Information Favourable Opinion