PNH National Services

Clinical Trials for PNH

Treatment for PNH has progressed significantly in the last 20 years. We are now in a position to look at optimising treatment for patients in terms of the modality of treatment and managing anaemia for patients experiencing extravascular haemolysis.

The National PNH Service has clinical trials currently open (Leeds, King’s and Monklands) for patients who are new to treatment and those already on treatment with Eculizumab or Ravulizumab. These include self-injection infusions or short injections, oral treatments or a combination of intravenous or oral treatments.

We aim to offer all patients with PNH who are eligible for a clinical trial option for them to consider, and this will be discussed at clinic appointments.

Trial NameTrial TargetMethod of ApplicationLeedsLondonMonklands
ALXN 2040-301Addition of Danicopan to C5 inhibitionOral and intravenous Ravulizumab or EculizumabClosedClosedClosed
ALXN 2050C5 inhibitor treated patients – proximal complement inhibitorOralN/AOpenN/A
Commodore 1Naive patients – C5 inhibitor trialSubcutaneous injectionClosedClosedN/A
Commodore 2Eculizumab treated patients – C5 inhibitor trialSubcutaneous injectionN/AOpenN/A
Regeneron 20105Eculizumab treated patients – C5 inhibitor and SiRNASubcutaneous injectionClosedN/AN/A
Novartis ApplyEculizumab treated patient – proximal complement inhibitorOralClosedClosedN/A
Biocryst 202C5 inhibitor treated patients – proximal complement inhibitorOralClosedClosedN/A
Regeneron 2021Naive patients РC5 inhibitor and SiRNA Subcutaneous injectionOpenN/AN/A
OmerosC5 inhibitor treated patients – proximal complement inhibitorIntravenousOpenN/AN/A


Prevention of Meningococcal Infection: Vaccination and Antibiotics

Individuals treated with anti-complement therapies are at increased risk of infection by a specific bacterium (Neisseria Meningitidis). Both vaccinations against Neisseria Meningitidis and preventative antibiotics are used to reduce this potential risk.