Professor Ranganath, the chief investigator of DeveleopAKUre, explains where we are with SONIA 2, the importance of SOFIA and why he enjoys working with AKU patients from all over the world.
Now the 3rd visits are over for SONIA 2, what progress can you report? What are the next steps?
All visits 3 have now been completed at all 3 study sites (Piešťany, Paris, and Liverpool). All tests that have been carried out have been analysed and assessed and are being entered into electronic records. Once this is complete, all data will be checked for reliability. Statisticians will analyze the data and decide if the study objective (to lower HGA) has been met. Then a report will be produced.. Once the report for the 1-year analysis has been finalised, the plan is to communicate some of the results to all participants. This will take the form of either a letter sent to all patients, or displayed on the study websites, and most likely both. The study will continue for a further 3 years, independent of the outcome of the analysis, as the European Medicines Agency wants to see the effect and safety of nitisinone in AKU over a longer period of time than just 1 year
What do you hope to achieve with the SOFIA study? – Why is it important to patients?
The SOFIA study is important as we do not know at present when to start treating AKU patients with nitisinone. The damage in AKU is mainly due to formation of a brown/black pigment. We can see this pigment in people with AKU in the eyes and ears by around age 30 years. What we do not know is if this pigment starts to form earlier in life before it becomes visible to the naked eye. If we can see pigment under the microscope in an ear cartilage tissue sample obtained by biopsy, this will then tell us at what age pigment starts to appear; this will then let us decide whether to use nitisinone in AKU patients before that age or not. This is why SOFIA is so important. We are looking at age groups as low as age 16 years; if we find pigment in 16 year olds then there will be a justification for more research in children.
What do you enjoy most about working with AKU patients and DevelopAKUre?
I really value working with people where my contribution will hopefully make a difference to their lives. The patients are such a mixed and interesting bunch of people who value what myself and others are doing for them and it makes it all worthwhile. When we are successful in getting the European Medicines Agency to license nitisinone for people with AKU, it will bring hope to thousands of people that there is a disease modifying treatment for this condition for the very first time. I absolutely love working in DevelopAKUre as this is a fantastic enthusiastic group of people who have come together and dedicated themselves to ensure that nitisinone treatment becomes available to all.
Can you introduce Dr. Milad Khedr and explain his role?
The clinical team in Liverpool delivering SONIA 2 and SOFIA includes doctors, nurses, administrative staff and researchers. There has been a new addition to the medical team with the appointment of Dr Milad Khedr who is starting his research into why tyrosine is high after nitisinone when used in AKU. Besides clarifying the nitisinone treatment, he will also be helping both SONIA 2 and SOFIA by being a study doctor.
DevelopAKUre is a series of major international clinical trials, run by a consortium of 12 European partners. It aims to study a potential new drug, called nitisinone, and assess its potential effectiveness in treating the rare disease, alkaptonuria (AKU).
DevelopAKUre is co-funded by a grant from the European Commission. This website is run by a UK patient group, the AKU Society. Learn more about AKU on the AKU Society's What is AKU page.
In today’s blog Chief investigator Prof. Ranganath reinforces the need for SONIA 2 pat.. ...more