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European first for Nottingham patients with rare kidney disease
Renal research specialists based at Nottingham City Hospital proved quick off their marks in supporting pioneering research into a chronic kidney disease.
They have randomised the first patient in Europe into a study investigating the impact of a new type of drug to slow the progression of Alport Syndrome. This is a rare and serious genetic condition which damages patients’ kidneys, as well as causing hearing loss and eye abnormalities.
The Renal Research team were quick off the mark in terms of recruiting the first patient because they received the ‘green light’ to start the study on March 4th – before the first COVID-19 lockdown hit the UK.
“We were ahead of the game in setting up the study,” said Dr Matt Hall, Consultant Nephrologist and Principal Investigator at NUH for the Alport study (pictured).
“Alport Syndrome also has a very active patient support group who are always informing members about research activity such as clinical trials.”
As well as Dr Hall, the NUH ‘Team Renal’ member who was instrumental in getting the study off the ground was Dr Sarah Brand, senior research nurse in the renal and transplant unit, who is study co-ordinator and administered the new drug to the first patient volunteer.
The female patient, who lives in Nottinghamshire, received the first of a course of sub cutaneous injections as part of the Phase II study of experimental drug SAR339375 (also known as Lademirsen) as a new way of treating Alport Syndrome. Through an indirect route which involved her contacting the support group in the USA and a London-based professor investigating renal disease, the first patient was eventually informed that an Alport study was open at Nottingham City Hospital, close to her home.
It’s another breakthrough for the Renal Team at Nottingham University Hospitals in terms of supporting innovative research and enabling studies at NUH to continue for high priority patients throughout the COVID-19 pandemic.
Researchers based at 12 sites around the world – including another two in the UK – ultimately aim to recruit a total of 45 patients into the trial.
Other patients with this rare kidney condition are now being approached for their consent to join the trial over the coming weeks.
Just 100 patients in Nottinghamshire live with this permanent condition, which can present with changes in urine in childhood and declining kidney function from the early twenties in adults. Alport Syndrome leads to scarring of the kidney and, ultimately, the need for the patient to undergo dialysis and potentially kidney transplants.
There is no specific, proven treatment yet for the disease but researchers are looking for new interventions to slow the progression of kidney damage.
This would mean the patient would benefit from years – or even decades – without the need for dialysis. Enabling good control over blood pressure in patients is one way of achieving this, along with healthy lifestyles.
The Alport drug trial is aimed at developing a brand new type of therapy that reduces inhibitory micro-RNA. A theory under investigation is that use of the new drug could dampen down genetic activity in the kidneys which leads to scarring.
If the new drug achieves the desired results, patients could ultimately receive it along with usual blood pressure medications.
Dr Asela Bandara, Medical Advisor at trial sponsors Sanofi, said:
“NUH’s contribution to this international trial has been instrumental in getting this programme off to a very positive start despite the challenges posed by the pandemic. The research team’s pragmatic and collaborative approach to setting up and recruiting for the trial has set an example to fellow colleagues in the UK and rest of Europe.”