Patients with Idiopathic Intracranial Hypertension (IIH), which causes headaches and potential sight loss, are being invited to take part in new research investigating the impact of weight loss drugs on their condition and eye health.

The IIH Advance trial, coordinated by clinical academics at BHP founder-member the University of Birmingham, aims to recruit 86 people with IIH from around the UK to take part in the trial lasting over a year, which will be delivered in partnership with Specsavers.

IIH is currently considered a rare disease with, affecting approximately 5,000 patients in the UK. It predominantly affects women of childbearing age, and 90% of those living with it also experience obesity. Previous studies at the University have established a direct link between IIH, metabolic dysregulation and obesity, so researchers anticipate that IIH prevalence is set to increase significantly as obesity levels continue to rise.

During the trial, participants will receive the weight loss drug Tirzepatide (commonly known as Mounjaro) and will have Optical Coherence Tomography (OCT) eye scans at a participating branches of Specsavers to monitor swelling of the optic nerves, called papilloedema, which is caused by intracranial pressure.

The study is delivered through the National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre (BRC).

Dr Maria Lange from the University of Birmingham and co-investigator on the trial said: “IIH is a debilitating condition, and after years of research to better understand the condition, this innovative trial will see whether weight loss, achieved by using the drug Mounjaro could have a significant positive impact for patients.”

“As GLP-1 medicines such as Mounjaro have become available for weight management through the NHS, we hope that the IIH Advance trial will establish a link between losing weight using these drugs and reducing the symptoms of IIH.”

Welcoming the trial, Specsavers’ director of professional advancement Paul Morris said: “IIH is a serious condition that can lead to blindness, and the role that community optometrists and their skilled teams can play in harnessing technology to detect serious eye conditions is pivotal. That’s why regular sight tests are so important. We welcome this innovative trial and are looking forward to collaborating with participants in it as well as the team at the University of Birmingham.”

People with a diagnosis of IIH who have papilloedema and live in the UK will be eligible to take part and are invited to self-nominate themselves to the trial. No doctor’s referral is required, and patients will not need to visit hospital or the trial centre in Birmingham.

Each participant will receive regular deliveries of Mounjaro to their home and will have regular calls with a research team member. They will also undergo eye scans at a participating Specsavers practice at the start of the trial, at six and 12 months, and at the conclusion of the trial.

Dr Jessie Gew, from the University of Birmingham and co-investigator adds: “IIH patients can now directly contact the trial team to self-refer. Once contact is made, our team will work closely with each participant to review relevant medical documentation and confirm eligibility for the study.”

Professor Alex Sinclair, Consultant Neurologist and Professor of Neurology at University of Birmingham, who leads the  Idiopathic Intracranial Hypertension Clinical Service at University Hospitals Birmingham NHS Foundation Trust, said : “This trial is an example of the type of pioneering research that could ease pressure on the NHS through innovative community-focused collaboration with industry. We believe that this vision for a trial that streamlines care and empowers patients to participate and receive treatment from the comfort of their own homes is exactly the type of research that the NHS 10-year plan is calling for.”

IIH Advance is now open for participants to self-refer online.

The first participant has been treated in an early-phase clinical trial investigating whether a new medical device, Carbalive, can slow disease progression and improve outcomes for patients with a rare liver disease called primary sclerosing cholangitis (PSC). The trial, known as the CATCH trial, marks the first time this device will be studied in humans with PSC-IBD.

The new clinical trial involves several BHP partners – it’s being coordinated by researchers from the University of Birmingham and the National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, and clinicians at University Hospitals Birmingham (UHB) NHS Foundation Trust. 

PSC affects around 3500-4000 people in the UK, including both adults and children. In PSC, the body’s immune system attacks itself, causing inflammation and scarring of the bile ducts – the small tubes that carry bile (a digestive fluid produced by the liver) to the gallbladder and small intestine. This leads to reduced or blocked bile flow, and over time, liver damage.

In around 80% of people with PSC, the body also attacks the bowel, which can lead to inflammatory bowel disease (IBD), a devastating disease that affects the lining of the gut, leading to severe gut inflammation, bleeding and diarrhoea.

There is currently no cure or medication to improve survival, and liver transplantation remains the only life-saving option for PSC.

The exact cause of PSC is not known, but research has shown that gut inflammation, that is also a key component of IBD, is a key trigger for the disease. Removing harmful substances and restoring the disturbed gut microbiome reduces gut inflammation, which may help slow its progression.

Carbalive (also called YAQ001) is a medical device created by Yaqrit – a company that develops life-saving treatments for patients with advanced liver disease. This device is made up of tiny carbon beads with lots of different sized pores to absorb toxins that would otherwise return to the liver.

In pre-clinical studies, animals with cholestatic disease (a condition where bile flow from the liver is impaired) showed improvements in liver function and signs of disease activity after treatment with Carbalive. Early studies in people with cirrhosis have also shown the device to be safe and effective in restoring the gut microbiome and reducing inflammation.

Palak Trivedi, Principal Investigator of the study, Consultant Hepatologist and Clinician Scientist at UHB, and Professor of Cholestatic and Immune-mediated Liver Disease at the University of Birmingham, said: “PSC-IBD is a progressive condition, and with no approved treatments, it can ultimately lead to liver failure.

“This trial is an important step in exploring the potential of Carbalive to slow the progression of this disease and improve patient outcomes. By funding this trial, and this innovation, LifeArc offers hope to patients and their families through a promising new approach.”

First participant in the trial

Nick Haynes, from Nottingham, is the first participant to take part in the trial and began his treatment this week.

Nick said: “I was diagnosed with PSC a couple of years ago, and as there aren’t many treatment options available currently, that really attracted me to taking part in this trial. I think it comes with many benefits – you hope the medicine will lead to some kind of improvement, but it’s also very reassuring to be part of enhanced screening. It’s about looking to the future too, and being part of something that could advance medicine and treatment options for people with PSC feels really positive. I really hope it becomes a viable treatment for patients in the future.”

Twelve patients with PSC-IBD will receive Carbalive orally for twelve weeks, and the data collected will assess whether the treatment improves bile flow and reduces inflammation.

The trial has been made possible through funding from LifeArc, a not-for-profit organisation supporting research into rare diseases.

Troels Jordansen, Yaqrit’s Chief Executive Officer said: “This is a great opportunity for Yaqrit to help patients with PSC-IBD while remaining highly focused on the treatment of late-stage liver disease.

“In YAQ001, the company has developed a treatment that addresses toxicities and inflammation that are common in advanced liver disease but also apparent in a broader spectrum of conditions.”

Sam Barrell, CEO of LifeArc, said: “For the 3.5 million people in the UK living with rare diseases, access to treatment is a huge challenge – 95% of conditions have none. That must change. World-first trials, like the CATCH Trial, are part of the solution, which is why LifeArc funded it through our Pathfinder Programme. We have an opportunity to make the UK the go-to place for innovative trials like this, but to realise this ambition we need to transform how we support rare disease research, making a difference for rare disease patients much faster.”

CATCH is the latest clinical trial delivered through the NIHR Birmingham Biomedical Research Centre, a partnership between the University of Birmingham and University Hospitals Birmingham NHS Trust, which focuses on inflammatory disease research. Collaborative research from the NIHR Birmingham BRC has already seen nearly 1,000 clinical trials initiated and has informed UK clinical guidelines.