APPLY-PNH

• Research type

  Research Study

• Full title

  A randomised, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody

• IRAS ID

  290285

• Contact name

  Austin Kulasekararaj

• Contact email

  austin.kulasekararaj@nhs.net

• Sponsor organisation

  Novartis Pharma AG

• Eudract number

  2019-004665-40

• Clinicaltrials.gov Identifier

  NCT04558918

• Clinicaltrials.gov Identifier

  N/A, N/A

• Duration of Study in the UK

  2 years, 0 months, 21 days

• Research summary

  Research Summary: This is a phase 3 study to determine whether LNP023 is efficacious and safe for the treatment of Paroxysmal Nocturnal Haemoglobinuria (PNH) in patients who are still presenting residual anaemia despite treatment with anti-C5 antibody therapy. This is a randomised study comparing the treatment of LNP023 (62% chance of receipt) with patients continuing their current anti-C5 therapy. Patients will be followed for a treatment phase (part 1; 24 weeks) and an extension phase (part 2; 24 weeks). Patients initially randomised to receive standard of care will be able to switch to treatment with LNP023 for part 2. The study aim is to demonstrate superiority of LNP023 through the achievement of sustained increase in haemoglobin levels (≥ 2g/dL) and through achieving sustained haemoglobin levels of ≥ 12 g/dL, in the absence of red blood cell transfusions. Additionally the study aims to demonstrate safety and tolerability of LNP023 and increase in patient wellbeing.

  Summary of Results: Lay summary posted on ClinicalTrials.gov
  https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Fu2790089.ct.sendgrid.net%2Fls%2Fclick%3Fupn%3DXv3JSvJ-2B3M71ppf7N9agbRehJ-2Fi4xyo44sEgJVCl5BfgYnnnOYur7Ot9AlGhAUzXzBIX3FvhXfzW2FkDc6uVx4RKcbfyJ0iP-2Fy6CStt2xk8-3DOJcM_E1aO2-2BZlVOSJJV-2FajQqskegTd6IRomHYTi-2Fbt8SH3YIqR87SbAnavmGKrVOablrJEMGzHGpRF8XwQAvZYe6IN2-2FhZ3EueYT17j18co09v1B6Lsq0TyoRls8Sds3yXD9vWl3Zpu37FRF-2B9rCUkmMTMUO0TAsoGXb2W9v9KZywpFbsq7gmwF8C6325EX7b0FHp10b1g6FdJ-2FcMixFt4csqsQ-3D-3D&data=05%7C02%7Cwestminster.rec%40hra.nhs.uk%7C7d3d5928d85c4485a4fc08dc11fbac66%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638405019822897705%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=XZ%2F04NbIE%2F7%2B0eMZKteuPoNkFF9KgIogw2issZvmqNE%3D&reserved=0

• REC name

  London - Westminster Research Ethics Committee

• REC reference

  20/LO/1275

• Date of REC Opinion

  21 Jan 2021

• REC opinion

  Further Information Favourable Opinion