ENCEPH-IG Trial: The Challenges Of Running A Rare Disease Trial

Autoimmune encephalitis is swelling of the brain caused by the immune system attacking the body in error. It can cause people to become confused, drowsy, exhibit changes in behaviour, and have seizures. It affects around 1 in 100,000 people per year in the UK and because of its rarity and varied symptoms, receiving a diagnosis can be a lengthy process.

Delays in diagnosis of autoimmune encephalitis, and the lack of specific targeted treatment, have a negative impact on patient’s recovery. Thankfully, there is lots of ongoing work to raise awareness of the disease and research trials, like the Enceph-IG trial, are working to identify better treatment options. The Enceph-IG trial is looking to determine if intravenous immunoglobulin (IVIG) can help improve recovery time.

Running a trial in a rare disease area is challenging, but even more so these days with the ongoing impact of COVID-19 on research in the NHS. In most other trials, many hospital sites can take part because the condition is much more common. But for rare diseases like autoimmune encephalitis, the pool of hospitals is much smaller due to the requirement of staff to have the appropriate disease-related knowledge to be able to treat the condition. Even though the Enceph-IG trial is working in a rare disease area, we are actually looking for hospitals that may not regularly see patients with autoimmune encephalitis. We have worked hard with doctors and other healthcare staff to make sure the skills and knowledge needed to deliver the trial are those that lots of clinical staff will have.

Another challenging element of doing research in a rare disease area is that patients with autoimmune encephalitis are few and far between. This means it might be weeks or even months between potential participants presenting at a hospital. This can make it really tricky to identify patients who are suitable for the trial, and it makes it easy to forget all the different trial activities.

To help solve this problem, we have encouraged sites to raise awareness of the trial by displaying posters and by attending regular meetings at their hospital. Some sites have had great success working with the local lab to set up alerts for when someone requests a test that might mean there is a patient with autoimmune encephalitis. We have also developed short guides to remind everyone of the different activities they need to do.

We are also doing our best to provide lots of opportunities for hospital staff to learn more about research and autoimmune encephalitis. We host monthly meetings where we discuss the trial and share information about autoimmune encephalitis. In the past we have helped to support clinical staff to attend the annual Encephalitis Society Conference. The trial is also part of the NIHR associate PI scheme. The Associate PI scheme gives non-research-based healthcare staff an opportunity to get involved in a large multicentre trial and helps provide a bit more support to research staff.

Can you help?

The condition is rare but has a severe impact on those affected. The research team is currently seeking more hospitals to join the trial. The Chief Investigator is Professor Tom Solomon from the University of Liverpool’s Brain Infections Group.

If you would like to know more about the study or register as a potential site, please contact encephig@cardiff.ac.ukor visit Enceph-IG – Centre for Trials Research – Cardiff University. You can also follow the trial on Twitter @EncephIG