These will determine if a potential new drug, called nitisinone, can help patients with alkaptonuria (AKU).
The DevelopAKUre programme 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 AKU. Nitisinone is not licensed for AKU, but has been used by many patients off-label and at the National Alkaptonuria Centre in Liverpool. Those patients seem to experience benefit, but the only way to be certain a drug works is through clinical research. If this is successful, we will apply to get the drug licensed as a treatment for AKU.
The trials have been split into 3 parts:
- SONIA 1 (Suitability of Nitisinone In Alkaptonuria 1) was designed to ascertain the correct and most effective amount of nitisinone patients should take to reduce levels of homogentistic acid (HGA) – this is called dosage.SONIA 1 finished In June 2014 and the main scientific paper was published in December 2014. It found that levels of HGA are reduced by nitisinone and this is dependent on dosage levels.
- SONIA 2 (Suitability of Nitisinone In Alkaptonuria 2) is designed to test the safety and whether nitisinone slows down the damage HGA does to the body of AKU patients.SONIA 2 has fully recruited 138 patients over 3 trial sites in Liverpool, UK; Paris, France and Piešťany, Slovakia. The trial will take place over 4 years and will require patients to make up to 6 trips to their nearest trial site. The patients are split into two groups of 69 with one group taking nitisinone and one not taking nitisinone. This is important as it allows us to show the positive effect of nitisinone.
- SOFIA (Subclinical Ochronotic Features in Alkaptonuria) is designed to compare AKU patients from different age groups to study when symptoms start to present. The study enables us to understand at what age AKU patients need to start taking nitisinone to best combat the damaging effects of the disease.SOFIA will test patients for Ochronosis, the process where cartilage is discoloured black by the effects of AKU. This is thought to be the earliest sign of the more damaging symptoms of AKU. SOFIA aims to recruit 32 patients from a range of 7 age groups from 16 to 50+. The study will require one visit to the Royal Liverpool University Hospital.
We are still actively seeking patients for the SOFIA study, if you are interested in taking part or learning more, please contact Ciarán Scott by emailing [email protected]. Further information can also be found on our clinical trials microsite, www.developakure.eu.