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 Name||Trial Target||Method of Application||Leeds||London||Monklands|
|ALXN 2040-301||Addition of Danicopan to C5 inhibition||Oral and intravenous Ravulizumab or Eculizumab||Closed||Closed||Closed|
|ALXN 2050||C5 inhibitor treated patients – proximal complement inhibitor||Oral||N/A||Open||N/A|
|Commodore 1||Naive patients – C5 inhibitor trial||Subcutaneous injection||Closed||Closed||N/A|
|Commodore 2||Eculizumab treated patients – C5 inhibitor trial||Subcutaneous injection||N/A||Open||N/A|
|Regeneron 20105||Eculizumab treated patients – C5 inhibitor and SiRNA||Subcutaneous injection||Closed||N/A||N/A|
|Novartis Apply||Eculizumab treated patient – proximal complement inhibitor||Oral||Closed||Closed||N/A|
|Biocryst 202||C5 inhibitor treated patients – proximal complement inhibitor||Oral||Closed||Closed||N/A|
|Regeneron 2021||Naive patients – C5 inhibitor and SiRNA||Subcutaneous injection||Open||N/A||N/A|
|Omeros||C5 inhibitor treated patients – proximal complement inhibitor||Intravenous||Open||N/A||N/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.