This year marks the end of a ten year long clinical research trial into a ground-breaking drug for renal patients and the culmination of an almost ‘unique achievement’ in clinical trials.

Researchers at University Hospitals Birmingham NHS Foundation Trust (UHB) are delighted that clinical trials they played a key part in, are now making a new drug available for people suffering from a life threatening kidney disease.

The Renal Research team at UHB was a big recruiter of patients to the first multi-centre trial of its type, leading to the use of Tolvaptan (JYNARQUE^TM/JINARC^TM), which is produced by Japanese company, Otsuka.

In 2015 the drug was approved for use in Japan, the EU (Inc. the UK), Canada, South Korea, Switzerland, Hong Kong, Australia, Turkey and Taiwan. It received its U.S. Food and Drug Administration (FDA) approval at the end of April 2018.

The Birmingham team recruited eleven patients into the trial, which ran between 2008 and 2018. The Principal Investigator being UHB Consultant Nephrologist, Dr Lukas Foggensteiner and the co-investigators were Prof Charlie Ferro, Dr Matt Morgan and Dr Punit Yadav.

Tolvaptan is the only licensed drug currently available to treat Autosomal Dominant Polycystic Kidney Disease (ADPKD), which is a progressively debilitating genetic disease, characterised by the development of fluid-filled cysts in the kidneys and is a significant cause of end-stage renal disease. ADPKD also affects families across multiple generations, since a parent with ADPKD has a 50 percent chance of passing the disease on to each of their children.

Patients at UHB participated in the TEMPO 3/4, TEMPO 4/4 and TEMPO 211 trials over ten years. Senior Renal Research Sister, Lesley Fifer, said: “It’s very unusual to have research last so long and of the eleven people recruited ten years ago, six completed ten years as trial participants. This is a very slowly progressing disease, so the trials had to be done over a long time.”

Consultant Nephrologist, Dr Foggensteiner, who is also an Honorary Senior Lecturer at the University of Birmingham, said: “This is an extremely unusual timescale by research standards and it’s a key achievement for the renal research team to manage a group of patients over such a prolonged period. It’s quite unique really and we are very proud to have played such a significant part.”

Currently, around 40 patients attending for renal treatment at UHB are benefitting from the use of Tolvapta, which should slow the progression of their disease, possibly helping them avoid dialysis and ultimately transplantation.