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Ground-breaking clinical trial offers treatment hope for IgA nephropathy

12 February 2024

A world-first clinical trial, designed around the insights of a kidney patient, has shown that the drug fostamatinib can reduce levels of protein in urine (proteinuria) – a common sign of kidney damage – in people with advanced IgA nephropathy (IgAN).

Trial marks first use of drug as a potential treatment for kidney disease

The SIGN clinical trial, led by Professor Fred Tam from Imperial College London and designed in close collaboration with a member of Kidney Research UK’s lay advisory group, marked the first use of fostamatinib in a clinical trial for kidney patients.

Results from the international pilot study, recently published in the influential journal: Kidney International Reports, showed that the drug, which is usually used to treat a blood disorder, was able to reduce levels of proteinuria by blocking an enzyme called ‘spleen tyrosine kinase’ (known as SYK).

This supported the findings of Fred’s earlier lab-based studies which suggest that SYK is involved in causing the inflammation and resultant kidney injury associated with IgAN, and stimulating the body to attack healthy tissue in other autoimmune diseases.

Work on the design of the SIGN clinical trial, which was funded by Rigel Pharmaceuticals, began in 2013. In total, 76 patients with IgAN, aged from 18 to 70, were recruited from five countries in Europe, Asia and North America, with the last patient completing treatment in 2018.

Participants were randomly selected to receive different doses of fostamatinib or a placebo tablet, twice a day for 24 weeks. All had persistent, significant proteinuria at the start of the trial, despite receiving the standard medication to protect their kidneys. But at the end of the trial, the group with the most severe proteinuria at the start showed a promising response to the higher dose of fostamatinib.

Patient involvement was pivotal to the success of the trial

“This was a collaborative international study with multiple researchers working together, but patient involvement from the outset was the key to its success,” says Fred.

“Thanks to Kidney Research UK’s lay advisory group I was introduced to a patient, Phil Smith, who has IgAN who joined us on the first day of our trial design meeting. His insights and input shaped the whole proposal.

“They advised us what would work and what wouldn’t for patients. And when we analysed the data, we noticed that patient compliance around taking the trial medication was very high, while the dropout rate was very low. In other studies, these rates have often been problematic.

Asian man sat in a lab, next to a microscope, wearing a white research coat
Professor Fred Tam

“Even if we had limitless funding, there are some things that money cannot provide – that’s why patient support and insights are priceless. Patients say things that, in hindsight are really obvious once they mention it, but as clinicians and researchers we wouldn’t have ever thought of it.

“This experience has completely changed the way I approach research now. Having patients suggest the key research questions and issues we should address in a clinical trial really is the way forward.”
Professor Fred Tam

Results pave the way for a larger clinical trial

Fred now hopes to use the evidence and learnings from this pilot proof of principle study to develop a larger, worldwide clinical trial of fostamatinib as a potential treatment for IgAN.

Fred said: “This study has shown that fostamatinib is safe to use for kidney patients and suitable for further investigation. So a longer term treatment period would be a good design for a larger clinical trial of the drug, involving a greater number of kidney patients with more advanced disease from a wider range of countries. “IgAN is a serious disease that affects people across the globe. With extensive collaboration and careful study design, together we may be able to eventually find an effective treatment.”

Patient advisor, Phil Smith said: “Thanks to Professor Fred Tam and his team, another important contribution has been made towards the goal of controlling and curing IgAN. Patients have always been at the centre of his research both in planning and evaluation and his innovative research, which repurposes an existing drug with positive outcomes, will save many millions of pounds in drug development and make access to the treatment much quicker.”

Elaine Davies, director of research operations at Kidney Research UK said: “At Kidney Research UK, we involve people living with and at risk of kidney disease in everything we do. We are delighted to see the impact that patient collaboration has had on Fred’s research  and we encourage more researchers to adopt this approach.”

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