A Phase 3 Study in Adults with PNH Who are Currently Treated with Eculizumab or Ravulizumab
Research type
Research Study
Full title
A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB
IRAS ID
1004724
Contact name
Karen Balangkig
Contact email
Sponsor organisation
Regeneron Pharmaceuticals, Inc.
Eudract number
2020-002761-33
Clinicaltrials.gov Identifier
Research summary
Paroxysmal nocturnal haemoglobinuria (PNH) is a chronic, progressive, life-threatening and rare multisystem disease. It is characterised by uncontrolled destruction of red blood cells, white blood cells and platelets. This leads to an increased risk of anaemia and blocked arteries which could cause heart attack or stroke.
The standard of care treatment for PNH is eculizumab which is an antibody developed to target a protein involved in the destruction process of the blood cells. This has shown to be effective in reducing patient’s need for blood transfusions, risk of blood clots and anaemia. Overall, it increases their quality of life and survival. Ravulizumab is an alternative standard of care therapy in the treatment of adult patients with PNH in several countries where it has been granted marketing authorization.
This is a Phase III eculizumab and ravulizumab controlled study to evaluate the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with PNH sponsored by Regeneron Pharmaceuticals, Inc. The aim of the study is to see how safe and effective the pozelimab and cemdisiran combination is for patients with PNH and how the combination compares with the existing treatments eculizumab and ravulizumab.
Approximately 140 participants will be randomized at a 1:1 ratio to the combination arm (pozelimab and cemdisiran) versus the standard-of-care arm (eculizumab or ravulizumab). The duration of the study including screening period to check eligibility for participation in the study (6 weeks), and treatment period (36 weeks) may be up to 42 weeks. Participants will receive the corresponding treatments according to their treatment assignment. The study participants will undergo multiple types of assessments, e.g. laboratory evaluations, blood test and transfusions, safety assessment etc.REC name
London - Westminster Research Ethics Committee
REC reference
22/LO/0233
Date of REC Opinion
21 Jun 2022
REC opinion
Further Information Favourable Opinion