PK and Safety study following single doses of Pozelimab and Cemdisiran

• Research type

  Research Study

• Full title

  AN OPEN-LABEL PARALLEL-DOSE STUDY OF THE PHARMACOKINETICS, PHARMACODYNAMICS, SAFETY, AND TOLERABILITY OF SINGLE DOSES OF SUBCUTANEOUSLY AND INTRAVENOUSLY ADMINISTERED POZELIMAB AS MONOTHERAPY AND IN COMBINATION WITH SINGLE DOSES OF SUBCUTANEOUSLY ADMINISTERED CEMDISIRAN IN JAPANESE HEALTHY VOLUNTEERS

• IRAS ID

  299719

• Contact name

  Jorg Taubel

• Contact email

  j.taubel@richmondpharmacology.com

• Sponsor organisation

  Regeneron Pharmaceuticals, Inc.

• Eudract number

  2021-001794-23

• Duration of Study in the UK

  0 years, 5 months, 1 days

• Research summary

  Inhibition of complement component 5 (C5) is effective in treating numerous autoimmune conditions. This activity is measured using the complement haemolytic activity assay (CH50). Pozelimab is a monoclonal antibody that binds to
  and inhibits the function of C5. Cemdisiran is a small interfering RNA, which suppresses liver production of C5. Both medications reduced the levels of CH50.

  This is a phase 1, open-label, parallel-dose cohort study of the pharmacokinetic (PK), pharmacodynamic (PD), safety,and tolerability, of a single dose of Pozelimab, given with or without cemdisiran, in healthy adult subjects of Japanese descent, as below. Each cohort will contain six subjects.
  • Cohort 1: pozelimab 300mg SC, single dose on Day 1.
  • Cohort 2: pozelimab 30mg/kg IV, single dose on Day 1.
  • Cohort 3: cemdisiran 50mg SC, single dose on Day 1. Pozelimab 100mg SC, single dose on Day 29.
  • Cohort 4: cemdisiran 50mg SC, single dose on Day 1. Polelimab 100mg SC, single dose on Day 1.
  • Cohort 5 (optional): cemdisiran ≤100mg SC, single dose on Day 1. Pozelimab ≤200mg SC, single dose on Day 1 or Day 29.

  For cohorts with dosing only on Day 1, participants will be admitted to the study unit on Day -1, dosed on Day 1,discharged on Day 2, and followed up until Day 113. For cohorts with dosing on Days 1 and 29, participants will be admitted on Day -1, dosed on Day 1, discharged on Day 2, readmitted on Day 28, dosed on Day 29, discharged on Day 30, and followed up until Day 141.

• REC name

  London - London Bridge Research Ethics Committee

• REC reference

  21/LO/0370

• Date of REC Opinion

  14 Jul 2021

• REC opinion

  Further Information Favourable Opinion