Evaluation of pegcetacoplan in pregnancy in patients with PNH
Research type
Research Study
Full title
Evaluation of pegcetacoplan in pregnancy in patients with paroxysmal nocturnal haemoglobinuria
IRAS ID
369648
Contact name
Richard Kelly
Contact email
Sponsor organisation
Leeds Teaching Hospitals NHS Trust
Duration of Study in the UK
4 years, 11 months, 31 days
Research summary
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare blood disorder where red blood cells are destroyed in the body, leading to blood clots and a higher risk of death. In the UK, two specialised centres in Leeds and London manage most of the cases. Since 2007, the main treatment has been drugs that block a part of the immune system called the complement pathway. One such drug, eculizumab, has been shown to improve survival rates in PNH patients, bringing them closer to the average survival rates for the general population. Before this treatment, people with PNH typically survived 10-15 years after being diagnosed.
Pregnancy increases the risk of blood clots in PNH patients, and before eculizumab, 8-21% of mothers died from clot-related issues, while 4-9% of babies did not survive. Eculizumab has since become the standard treatment for pregnant women with PNH, with no maternal deaths in a study of 75 pregnancies.
However, even with eculizumab, about 27% of PNH patients still need blood transfusions because their red blood cells don’t last as long in the body. In pregnancy this can cause increased risk for both the mother and baby.
Pegcetacoplan is a new treatment that works earlier in the complement pathway and has shown promise in improving anaemia in PNH patients already on eculizumab. However, only two cases of pegcetacoplan use during pregnancy have been reported. The goal of the current study is to understand if pegcetacoplan can pass through the placenta or into breast milk when used by pregnant women and to monitor the pregnancy to make sure the mother and fetus remain well.
REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
26/YH/0074
Date of REC Opinion
17 Apr 2026
REC opinion
Unfavourable Opinion