A clinical trial is underway in Liverpool, bringing together NHS University Hospitals of Liverpool Group (UHLG), the University of Liverpool (UoL), and Drugs for Neglected Diseases initiative (DNDi), a medical research non-profit organization, in the effort to address visceral leishmaniasis – one of the world’s deadliest parasitic diseases.
Visceral leishmaniasis is a neglected tropical disease found in parts of Asia, East Africa, and Brazil. It is caused by leishmania parasites and transmitted through the bites of infected sandflies. Despite its impact, the disease has received limited attention in many regions.
The disease presents with flu-like symptoms, including fever, fatigue, and weight loss, and is almost always fatal if left untreated.
Current treatments require daily injections for up to 17 days which can cause immediate pain during the treatment, nausea, vomiting, as well as more serious complications.
This study is testing an oral drug (DNDI-6899) that could overcome these barriers by being easier to store, transport, and administered—potentially saving thousands of lives.
The trial is being conducted at the NIHR Liverpool Clinical Research Facility (CRF) at the Royal Liverpool University Hospital and is funded by Wellcome. It is also part of the work done at the University’s Centre for Experimental Therapeutics (TherEx). The centre aims to improve health by optimising existing drugs and developing new treatments.
Professor Richard Fitzgerald, Director of the Liverpool CRF and Principal Investigator of the study, and Professor Saye Khoo from the Centre for Experimental Therapeutics at UoL, are leading the trial to evaluate the safety and effectiveness of the new oral treatment.
Professor Fitzgerald said: ‘Visceral leishmaniasis claims thousands of lives every year, and current treatments are often inaccessible to those who need them most, particularly in low-income regions where healthcare systems are limited.
‘This trial offers hope for a more practical and effective solution, one that could reach those most at risk and save countless lives.
‘It also reflects the skills and expertise here in Liverpool, showing that we are at the forefront of developing new medicines for a disease that affects people on the other side of the world.‘
Professor Saye Khoo, Director of TherEX, University of Liverpool added: ‘By collaborating with DNDi, we aim to develop a treatment that can be stored in varying conditions, delivered directly to vulnerable communities, and administered easily—without the need for hospitalisation.‘
The trial is currently recruiting healthy volunteers from Liverpool and the surrounding areas, with several participants already having completed the screening process. The trial will take place over two weeks at the Liverpool CRF, where volunteers will be closely monitored by clinical specialists.
The CRF offers a 26-bed unit, consisting of two four-bed bays and 18 en-suite single rooms. Additional amenities include a TV lounge, accessible en-suite bathroom facilities, and Wi-Fi for remote work and internet browsing. Catering services are also provided throughout the duration of the stay.
Dr Fabiana Alves, Director of leishmaniasis programme at DNDi said: ‘Visceral leishmaniasis affects the poorest of the poor and is linked to climate change, which can lead to its expansion to new areas.
‘One billion people are at risk globally. An estimated 50,000 to 90,000 new cases occur every year – most of them in Eastern Africa, where half of those infected are children under 15. We urgently need better, effective, safe and patient-friendly medicines, and this Phase I trial in Liverpool is playing a critical role toward that goal.‘
Sabrina Lamour-Julien, Research Lead in Therapeutics at Wellcome, said: ‘Neglected tropical diseases, including visceral leishmaniasis, are debilitating diseases that disproportionately affect the poorest communities. Their absence from the global health agenda means there are limited treatment options.
‘This clinical trial is a great example of the progress already being made for visceral leishmaniasis, particularly with the focus on areas with the highest incidence of disease – providing hope for a treatment that is tailored specifically to benefit those most at risk. It is a step in the right direction; we need to continue advancing research and innovation if we are to eliminate these diseases.‘
About the University Hospitals of Liverpool Group
Our group of hospitals provide acute and specialist care to the people of Merseyside and beyond.
NHS University Hospitals of Liverpool Group was formed on 1 November 2024, born from a shared aim to improve the care we provide our communities.
We operate from four hospital sites: Aintree University Hospital, Broadgreen Hospital, Liverpool Women’s Hospital and the Royal Liverpool University Hospital, alongside a host of community services.
We are one of the largest employers in region, with over 16,800 colleagues who are dedicated to caring for our communities – from birth and beyond.
For the 630,000 people across Merseyside, we are their local NHS. We provide general and emergency hospital care, alongside highly specialised regional services that extend to more than two million people in the North West.
Aintree University Hospital is the single receiving site for adult major trauma patients in Cheshire and Merseyside, and hosts a number of regional services including an award-winning stroke facility. Broadgreen Hospital is home to several elective surgical, diagnostic and treatment services, together with specialist patient rehabilitation. Liverpool Women’s Hospital specialises in the health of women and babies, delivering over 7,200 babies in the UK’s largest single site maternity hospital each year. The Royal Liverpool University Hospital is the largest hospital in the country to provide inpatients with 100% single en-suite bedrooms and mainly focuses on complex planned care and specialist services.
About DNDi
The Drugs for Neglected Diseases initiative (DNDi) is a not-for-profit medical research organization that discovers, develops, and delivers safe, effective, and affordable treatments for neglected people. DNDi is developing medicines for sleeping sickness, leishmaniasis, Chagas disease, river blindness, mycetoma, dengue, paediatric HIV, advanced HIV disease, cryptococcal meningitis, and hepatitis C. Its research priorities include children’s health, gender equity and gender-responsive R&D, and diseases impacted by climate change. Since its creation in 2003, DNDi has joined with public and private partners across the globe to deliver 13 new treatments, saving millions of lives.
Media contact: media@dndi.org
About TherEX
The Centre for Experimental Therapeutics (TherEx) is a centre dedicated to advancing clinical research in therapeutics and experimental medicine at the University of Liverpool. Based within the Department of Pharmacology and Therapeutics and work in collaboration with the NIHR Liverpool Clinical Research Facility.
The centre is committed to optimising drug use, providing tools for safe prescribing, and managing drug interactions, particularly for patients with multiple medical conditions. It focusses on generating evidence for the best dosing practices in special populations where current guidance is lacking, and on accelerating the evaluation of new treatments through early-phase clinical trials, including first-into-human studies.
About NIHR Clinical Research Facility
The NIHR Liverpool Clinical Research Facility is a state of the art, purpose built, facility, opened in 2009 to perform clinical trials of new medications and treatments. Embedded within the Royal Liverpool Hospital which also provides specialist equipment, 24-hour emergency coverage and access to ITU, the CRF offers a safe and regulated environment to perform clinical research trials to the highest possible standards.
About Wellcome
Wellcome supports science to solve the urgent health challenges facing everyone. We support discovery research into life, health and wellbeing, and we’re taking on three worldwide health challenges: mental health, infectious disease and climate and health.
Photo credit: Mercy Mumo-DNDi
