An international clinical trial of a potential new treatment for Progressive Supranuclear Palsy (PSP) will soon start recruiting participants in the relatively early stages of the disease.
The phase II trial involves a new intravenous drug which is being developed by Biogen, a biotechnology company specializing in developing medicines for neurological conditions and rare genetic disorders. The drug is an antibody that is designed to target and remove tau, the protein that forms the tangles and clumps seen in diseased nerve cells in PSP.
The clinical trial will involve around 400 people with PSP at centres across the world and aims to establish the safety and efficacy of the drug. It is a placebo-controlled trial, meaning that half the participants will be given the drug while the other half will receive placebo, which looks the same but contains no active drug. Neither the participants nor the nurses and doctors at the trial centres will know who is taking the real drug; this is an essential part of ensuring the results of the trial are robust and that any beneficial effects can be reliably attributed to the new treatment.
Where will the trial take place?
At the moment, 8 UK centres for the trial have been confirmed: London (UCL), Cambridge, Glasgow, Newport, Newcastle, Brighton, Liverpool and Southampton. It is possible that more centres could be added at a later date.
Who can take part?
Each centre will be recruiting only a small number of participants, with a target of around 30 participants for the whole of the UK. In order to be considered for the trial, people with PSP will need to have experienced their first symptoms less than 5 years before enrolling and should still be able to walk independently or with minimal assistance.
The individual patient participation in the trail lasts just over a year. It is divided into a 6-week screening period (when all the necessary assessments will be carried out to check that you are eligible to participate), and a 52-week dosing period, (when either the study drug or placebo is given), with clinic visits approximately every 4 weeks. Once this portion of the trial concludes, there will also be an opportunity to participate in an open-label dosing period where everyone receives the study drug.
Participants will need to commit to monthly attendance at one of the trial centres, accompanied by a carer. Taking part in the trial will involve receiving intravenous infusions of the drug or placebo, along with a range of tests including MRI brain scans. If you have a pacemaker or other implant that prevents you from having MRI scans you will not be able to take part.
There are a number of other criteria that participants need to meet. A researcher at one of the trial centres will need to go through these with you before they can enrol you for the trial.
Where can I get more information?
You can contact the PSPA Helpline: 0300 0110 122 or email@example.com
Our clinical trials information sheet explains how trials work and describes some of the issues you might like to consider before deciding whether or not you would like to take part.