SONIA 2: Suitability Of Nitisinone In Alkaptonuria 2

Alkaptonuria (AKU) was first discovered in 1902. More than 100 years later, the drug nitisinone has been identified as the first potential treatment for AKU. Nitisinone is already licensed as a treatment for another rare disease, but it’s yet to be approved for treating AKU.

But SONIA 2, our second clinical trial, exists to try and change this. It will last four years and will assess the long-term suitability of nitisinone for use in AKU patients. Following the completion of SONIA 1, our first clinical trial, SONIA 2 began in 2014. We have successfully recruited all of the patients we need, and recruitment has now ended.

You can find out more about the different stages of the trial in this video from Professor Ranganath:

The trial is based at three test centres in Europe: Liverpool (UK), Paris (France) and Piestany (Slovakia). Patients will make a total of 6 visits to a test centre, with each visit lasting 2 to 4 days. They will also complete questionnaires between visits and there will be a follow up phone call a month after the last visit.

Patients have been randomly divided into two groups. One group will receive nitisinone and the other will receive no treatment. Comparing the two groups is essential to prove nitisinone slows the progression of AKU in order to get nitisinone licensed for AKU.

Both the treatment and non-treatment groups have regular contact with AKU experts. These experts and other health practitioners monitor the progression of AKU and general health through a range of assessments. For those in the treatment group, they will also monitor any side effects that may occur. To find out what the different assessments may involve, take a look at the videos available to watch here.

Long distance and international travel within Europe is arranged and paid for by the trial. Accommodation is provided at a local hotel. Other reasonable expenses such as local travel will be reimbursed. We have received funding from the European Commission to cover these costs.

All official printed information will be translated into patients’ native languages and interpreters will be provided for non-English speakers during test centre visits. Wherever possible, we will arrange for you to visit the test centre with another patient who speaks the same language as you.

We are conducting these clinical trials to help patients with AKU: nitisinone could be the treatment that they have been waiting for.