PRINCE
Research type
Research Study
Full title
A Phase 2, dose-ranging study in patients with Paroxysmal Nocturnal Haemoglobinuria (PNH), treated with Cemdisiran and Eculizumab.
IRAS ID
252932
Contact name
Anita Hill
Contact email
Sponsor organisation
University of Leeds
Eudract number
2019-000816-27
ISRCTN Number
ISRCTN31882639
Duration of Study in the UK
1 years, 3 months, 0 days
Research summary
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, clonal hematopoietic stem cell disorder of the blood characterized by the destruction of red blood cells by the complement system. Standard of care is treatment with eculizumab, a humanized monoclonal antibody. Eculizumab has been approved for use in patients with PNH for over 10 years and has a known efficacy and safety profile. However, significant clinical challenges remain. Patients receiving Eculizumab may still experience breakthrough haemolysis. Therapy is often lifelong and the regimen can therefore be burdensome with time off work or education.
The new treatment being tested in this study is called cemdisiran - this is a synthetic RNA interference (RNAi) therapeutic designed to suppress liver production of C5 protein. Cemdisiran will be given in combination with Eculizumab in cohorts of 3 patients at differing dose schedules over 32 weeks.
The main aim of the trial is to determine a sufficiently effective dose and schedule of cemdisiran in combination with eculizumab.
This is a single centre study recruiting PNH patients who are currently receiving Eculizumab at St James's University Hospital, Leeds.REC name
South Central - Oxford C Research Ethics Committee
REC reference
19/SC/0453
Date of REC Opinion
24 Feb 2020
REC opinion
Further Information Favourable Opinion