Research is important in order for new treatments to be developed to treat diseases.

Taking part in research is a personal choice and may also depend on whether you are eligible to take part in a particular research study. There are currently a number of clinical trials taking place in the United Kingdom for treatments for PNH.

To find out what clinical trials are taking place in which you may be eligible to take part, speak to your haematologist or Clinical Nurse Specialist at the PNH National Service. Alternatively you can look up clinical trials which are currently recruiting patients in the UK in the international Clinical Trials Register described below. In the search box, type “PNH” into “Condition or disease” and your country and city into the “Country” and “City” boxes. You may also want to check out the PNH trials taking place internationally on the PNH Global Alliance website here.

International Clinical Trials Register

This is a register for trials and their results for worldwide clinical trials (whether publicly or privately funded). The inclusion of adverse event information was optional when this database was first released in 2008 but this information has since been required from September 2009. Results of trials for registered and completed studies is submitted by the study sponsor (or principal investigator) in a standard, table format without explanations or conclusions. The information is considered summary information and does not include individual patient data.

European Register of Clinical Trials

This is a register of all interventional clinical trials on medicines conducted in the European Union or the European Economic Area which started after 1 May 2004. This is the database used by national medicines’ regulators. The results of these trials are entered into the database by the sponsors of the trials themselves and are published in this register once the sponsors have validated the data. For more information please see here

PNH Registry

The PNH Registry is an international research database which collects anonymised data about PNH patients. Participation is voluntary and all patients with a PNH diagnosis or clone are eligible to participate as long as they give informed written consent to take part. The PNH Registry is a what is called a ‘post marketing study’ and was set up following the licensing of Soliris for treatment of PNH patients to collect data to evaluate the safety of the medication and to understand the disease progression of PNH. It is owned and managed by Alexion who make Soliris and ravulizumab and which are required to report to regulators about long term safety of these drugs. The PNH Registry is known as a non–interventional study which means taking part does not affect a patient’s normal medical care.

When someone joins the PNH Registry they are asked to complete a health assessment questionnaire and which is repeated again at six monthly intervals at their outpatient appointments. Baseline data including demographic details, medical history, and PNH relevant medications are also collected.

Clinicians complete further forms every 6 months to document any development of disease, complications and hospital stays. Information will also be collected about any blood transfusions patients have received, blood test results, new or changes to medications and pregnancy.

The PNH Registry collects data about natural history, management of patients with PNH and safety data on those treated with eculizumab (Soliris) and ravulizumab (Ultomiris). Patients treated with eculizumab and ravulizumab will also have information collected about protection against meningococcal septicaemia, any reactions to the drug and the eculizumab/ravulizumab dosage.

The information collected by the PNH Registry will hopefully improve understanding of PNH and should help to improve treatments in the future. A patient representative from the PN H Global Alliance sits on the PNH Registry Executive Committee.