ACCESS-EXTENSION
Research type
Research Study
Full title
An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria
IRAS ID
1006175
Contact name
Danise Subramaniam
Contact email
Sponsor organisation
Regeneron Pharmaceuticals, Inc.
Eudract number
2021-004931-10
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 an antibody which targets a protein Complement C5 (C5). C5 is a substance generally involved in fighting infection in a body, but in patients with PNH, C5 is involved in the destruction process of the blood cells. Eculizumab is shown to be effective in reducing patient’s need for blood transfusions, risk of blood clots and anaemia. Ravulizumab is an alternative standard of care therapy in the treatment of adult patients with PNH in several countries where it has marketing authorisation.
This study is a phase 3 open-label, single arm, multicenter study evaluating the long-term safety, tolerability and efficacy of the combination of pozelimab and cemdisiran in patients who have been treated with the combination of pozelimab and cemdisiran in the following parent studies: R3918-PNH- 2021 and R3918-PNH-2022, sponsored by Regeneron Pharmaceuticals, Inc.
A combination of cemdisiran and pozelimab works together to reduce the levels and activity of C5. Therefore, the combination may improve signs and symptoms of PNH and potentially other diseases in which C5 is involved.
Approximately 288 participants will be enrolled, participants start of the 108-week open-label extension period (OLEP) in alignment with the last visit in the parent study (eg. End of Study visit for R3918-PNH- 2021/R3918PNH-2022). On day 1 of the OLEP, patients will begin extension treatment with pozelimab 400 mg + cemdisiran 200 mg, both administered subcutaneously every 4 weeks.
The study participants will undergo multiple types of assessments, e.g. laboratory evaluations, blood test and safety assessment etc.REC name
London - Westminster Research Ethics Committee
REC reference
22/LO/0793
Date of REC Opinion
12 Apr 2023
REC opinion
Further Information Favourable Opinion