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 which you may be eligible to take part in, speak to your haematologist or Clinical Nurse Specialist at the PNH National Service.
What is a clinical trial?
Here is a short video explaining what a clinical trial is:
Clinical Trials: Learning about the Facts and Myths
Eric Parsons from Cleveland Clinic talks about what you need to know about clinical trials. He addresses the types of clinical trials, what happens during a clinical trial, along with common misconceptions and answer frequently asked questions. Eric also shows you how to navigate clinicaltrials.gov and the AAMDSIF clinical trial spotlight page.
This is a register of all trials currently taking place in Europe. It contains information on 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 for data related to clinical trial protocols. The data on 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.
There is a registry and results register of worldwide clinical trials (whether publicly or privately funded). The submission of adverse event information was optional when this database was first released in 2008 but this information was required from September 2009. Results information for registered and completed studies is submitted by the study sponsor or principal investigator in a standard, tabular format without discussions or conclusions. The information is considered summary information and does not include individual patient data.
A biosimilar medicine mimics the effects of a previously approved biologic medicine and is intended to treat the same disease or diseases. Biosimilars should not be confused with traditional generic medicines, which are considered to be identical to the original medicine. Biosimilars are being developed for PNH based on understandings made possible through the development and use of eculizumab (Soliris) in PNH over the past decade. It is likely that several new biosimilar medicines will come to the market in the short to medium term.
Multiple research projects into PNH and bone marrow failure syndromes are taking place at the PNH National Service centres in Leeds (St James’s Hospital, Leeds and Kings College Hospital, London).
Professor Peter Hillmen from St James’s Hospital, Leeds discusses Future advancements in the treatment of PNH (2017)
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.
When someone joins the Registry they are asked to complete a health assessment questionnaire and then asked again at six monthly intervals at their outpatient appointments. Baseline data including demographic details, medical history, and PNH relevant medications are also collected.
Clinicians will 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 changed medications and pregnancy.
The Registry is non–interventional study which means taking part does not affect a patient’s normal medical care. The registry collects data about natural history, management of patients with PNH and safety data on those treated with eculizumab. Patients treated with eculizumab will also have information about protection against meningococcal septicaemia, any reactions to the drug and the eculizumab dosage collected.
The information collected by the Registry hopes to improve understanding of the disease and should help to improve treatments in the future.
The Registry covers 28 countries worldwide. At present over 2,500 patients with PNH have been included in the Registry. The United Kingdom is the second largest recruiting country having entered over 350 patients to date.