First patient recruited globally in Phase II clinical trial for Alport syndrome
Alport syndrome is a genetic condition that is identified by kidney disease, loss of hearing, and eye abnormalities. Currently, there are limited treatment options for Alport syndrome, leaving many patients at risk of disease progression.
Nottingham University Hospitals (NUH) is the first site to globally recruit a patient into a study investigating the effectiveness of a new medicine in patients with Alport syndrome. The National Institute for Health and Care Research (NIHR) is supporting the SETA101 study, assessing the safety, tolerability, and preliminary efficacy of Setanaxib. The success of the study will provide insight into the benefits of using Setanaxib alongside the standard treatment of care by preserving kidney function in Alport syndrome patients.
The study is supported by the Clinical Research Network (CRN) East Midlands, and sponsored by Calliditas Therapeutics AB in partnership with Medpace UK Limited.
The randomised, placebo-controlled Phase II trial is expected to enrol approximately 20 patients with a confirmed genetic diagnosis of Alport syndrome. The trial will evaluate Setamaxib over 24 weeks whilst also evaluating kidney function by measuring urine protein and certain markers in the blood and urine compared to placebo.The study will be conducted at multiple sites across the UK.
Sarah Brand, Renal Research Team Leader at NUH provides us with more information.
Why is the study important?
Alport syndrome is a rare condition which can result in impaired renal function. It is an inherited condition, but one for which the prevalence is widely acknowledged to be unknown. It commonly causes the deterioration of renal function. The progression of the disease is variable, and this variation in prognosis and severity is largely believed to be associated with genetics and the different patterns of inheritance. There is a wide range of progression ranging from slow kidney function decline, to rapid disease progression to end-stage renal disease in adolescence or early adulthood. At present there is no curative treatment for Alport syndrome. This study is investigating the safety, tolerability and efficacy of setanaxib in patients with Alport syndrome. This is crucial to broaden the potential treatments for this condition, given that currently only symptom management is possible. This study is recruiting from the paediatric and younger adult population (up to age 40), thereby targeting those who may benefit most in terms of living long term with this potentially progressive condition.
How did you recruit the first participant?
We screened our patient database for those with a diagnosis of Alport syndrome, and identified those who appeared to be broadly eligible. They were all contacted personally by the Principal Investigator (PI) who is part of the usual care team and who introduced the study to them; this was followed by the approved patient information sheet. All patients were then contacted by the research nurse team who gauged interest in the study and answered any questions. Once patients expressed an interest, initial screening visits were arranged as soon as possible, to ensure that screening was expedited. Visits were booked in a flexible way to ensure that timings suited the potential participants as much as possible.
Did you face any challenges?
The patient pool is limited due to the rare nature of the disease, but having a robust searchable database of patients helped to easily identify potentially eligible patients.The first patient recruited required multiple contacts with the research team to allow time for her to fully read the patient information leaflet, so it required tenacity on the part of the research team to continue to communicate with the patient until they were able to make a decision. It was important for the patient to feel supported, but not pressured and this is sometimes a fine line to tread. The patient has a busy personal life, so the research team has been especially flexible with visit dates and times to accommodate the needs of the patient.
What would you say to someone thinking about taking part in research?
The patient recruited is from a diverse background and we have worked with her to explore her perceptions of research participation and feel the responsibility of ensuring that she has a good experience of participation in order for her to advocate to others in her community. We firmly believe that research is the way to improve patient care, and know that it is our responsibility as research teams to facilitate the opportunity to participate in research for as many patients as possible. As an inherited condition, taking part in research may directly influence the treatments which are available in the future for any affected children or grandchildren of potential participants. As it is also a rare condition, the pool of potential participants is very limited and therefore participation cannot be left to someone else. If anyone is considering participating in a study for Alport syndrome, you may be one of very few people who can assist in answering clinical questions about this rare condition
If you would like to find out more on how you can Be Part of Research, click here.
Find more information about the Setamaxib Phase 2 trial on Calliditas Therapeutics here.
