A SINGLE ARM, OPEN-LABEL STUDY TO ASSESS THE SAFETY, EFFICACY, AND PHA

• Research type

  Research Study

• Full title

  A Single Arm, Open-Label Study to Assess the Safety, Efficacy, and Pharamcodynamic Effects of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

• IRAS ID

  295375

• Contact name

  Richard Kelly

• Contact email

  richardkelly@nhs.net

• Sponsor organisation

  Regeneron Pharmaceuticals, Inc.

• Eudract number

  2020-005006-25

• Duration of Study in the UK

  2 years, 0 months, 22 days

• Research summary

  The purpose of this study is to research an experimental combination treatment with pozelimab and cemdisiran focused on patients with PNH (rare immune disease that causes red blood cells in your body to break apart). A combination of cemdisiran and pozelimab works together to reduce the levels and activity of C5 which may improve signs and symptoms of PNH. The study is being conducted to understand how effective the study drugs are at controlling the signs and symptoms of PNH, the side effects that can happen from taking the study drugs, and check to see if they are well tolerated in patients who switch from eculizumab treatment.
  This study will include about 12 people in one center in the United Kingdom. Patients will be men and women 18 years of age or older with a diagnosis of PNH, and currently on treatment with stable eculizumab therapy for at least 12 weeks prior to screening. Subjects will be in the main part of the study for about 32 weeks. If during the study, the PI decides to change a subjects dosing schedule, they will be asked to stay in the study on the new schedule for a period of 32 weeks starting from the day they start the new schedule. This means that the main part of the study will be longer than 32 weeks depending on when the new schedule is started. Once the mail part of the study is complete, there is an option to continue to receive the study drugs for 1 more year in an optional long-term extension of study. If patient opts out of the extension period the PI will monitor the patient for approximately 1 year after the last dose of study drugs.

• REC name

  Yorkshire & The Humber - Leeds East Research Ethics Committee

• REC reference

  21/YH/0047

• Date of REC Opinion

  15 Apr 2021

• REC opinion

  Further Information Favourable Opinion