Safety and effects of pozelimab and cemdisiran in patients with PNH

• Research type

  Research Study

• Full title

  A Randomized, Open-label, Two-arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy

• IRAS ID

  295782

• Contact name

  Richard Kelly

• Contact email

  richardkelly@nhs.net

• Sponsor organisation

  Regeneron Pharmaceuticals, Inc.

• Eudract number

  2020-005005-17

• Duration of Study in the UK

  3 years, 0 months, 15 days

• Research summary

  Research summary:

  The study is researching an experimental combination treatment with pozelimab and cemdisiran (called “study drugs” in this form). The study is focused on patients with paroxysmal nocturnal haemoglobinuria (PNH).

  PNH is a rare immune disease that causes red blood cells in the body to break apart.

  The aim of the study is to understand the side effects that can happen from taking the study drugs and check to see if the study drugs are well tolerated in patients with PNH.

  Participants will be in the main part of the study for about 29 weeks. If, during the study, participants study doctor thinks it would be best for them to change their study dosing schedule, participants will be asked to stay in the study on the new schedule for a period of 28 weeks starting from the day participants start the new schedule. This means that the main part of the study will be longer than 29 weeks depending on when the new schedule is started.

  After participants complete the main study period or, if it was required, the intensified treatment period, participants can choose to continue receiving the study drugs in an optional extension period.The optional extension period will end after 1 year, even if participant need intensified treatment during the optional extension period.
  Participants who stop taking the study drugs for any reason (including if they complete a study period [main study or optional extension period], if the Sponsor decides to end the study, and other reasons),will be monitored by study doctor for health check as the study drugs slowly leave participant's body. Participants will have study visits every 2 to 4 months where study doctor may check on their health, physical examination, check medications,and take blood samples.

  This study will include about 24 participants worldwide.

  Summary of results:

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• REC name

  Yorkshire & The Humber - Leeds East Research Ethics Committee

• REC reference

  21/YH/0096

• Date of REC Opinion

  12 Jul 2021

• REC opinion

  Further Information Favourable Opinion