As most of you will know, we launched clinical trials called DevelopAKUre in 2012. These trials are based in three sites around Europe (Liverpool, UK; Paris, France and Piestany, Slovakia) and will assess the drug nitisinone to test whether it is an effective treatment for AKU.

DevelopAKUre is made up of three parts:
1)  SONIA 1: a clinical study to compare different doses of nitisinone to see which dose provides the best effects.
2)  SONIA 2: a clinical effectiveness study, comparing a group of patients taking nitisinone against a group of patients not taking nitisinone. After 4 years, we can determine if nitisinone was beneficial in treating AKU.
3)  SOFIA: an observational study comparing a younger group of patients to an older group of patients to help us decide which age is the best to begin treating AKU.

Last year was important for DevelopAKUre as it saw the end of SONIA 1, the start to SONIA 2 and a year full of other developments.

Ending SONIA 1 meant the project board could analyse the data taken so far and decide on the best dose of nitisinone to study further. Their decision was eventually published as an academic paper and summarised on the DevelopAKUre site here. The dose chosen was 10mg of nitisinone daily, and is the dose currently being studied in SONIA 2. However, no changes will be made to the dose given at the National AKU Centre at the moment, until more data from SONIA 2 is gathered.

2014 also saw the start of SONIA 2. The lead centre for the study is the Royal Liverpool University Hospital, who last week sent out a press release: “Weed killer clinical trial is set to make the grass greener for black bone patients”. We aim to recruit 140 patients for SONIA 2, and have been seeing European patients since June. Recruitment is due to end in a few weeks’ time, so look out for more updates soon.

SONIA 2 will run for 4 years, collecting data throughout and only after that data has been analysed will we know for sure whether nitisinone is an effective treatment for AKU. However the signs so far do look hopeful.

The lead doctor, Prof Ranganath says, “Early findings of the 60 month clinical trial have shown the treatment has reduced the amount of homogentisic acid in patients’ urine by 98.8% – this means it’s nearly normal and their urine colour returns to yellow as opposed to being black. The early results of this trial indicate this treatment works and should, in time, pave the way for a cure for AKU patients”.

For more regular updates on DevelopAKUre, please look at the trial blog: DevelopAKUre blog. For any patient queries about the trials, please contact Lesley Harrison on