SONIA 2

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. We’re now looking for patients interested in taking part. Following the completion of SONIA 1, our first clinical trial, SONIA 2 is due to start in late 2013.

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

Patients will be 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 will have regular contact with AKU experts. These experts and other health practitioners will monitor the progression of your AKU and your 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 will be arranged and paid for by the trial. Accommodation will be provided at either the hospital or at a local hotel if necessary. 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. If you are interested in participating in this clinical trial or if you would like more information, please register your interest below and a member of the DevelopAKUre team will be in touch with you shortly.