The Next Step in Developing A Cure for AKU

Source: AKU Society 


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.

Lab experiments show nitisinone could stop the progression of AKU while clinical research from the US showed it can reduce levels of homogentisic acid (HGA), the cause of AKU damage, by up to 95%. However, further clinical trials are required to prove that nitisinone is effective in treating AKU.

Our first clinical trial, SONIA 1, is now almost complete and we are seeking AKU patients from across Europe to participate in our second longer trial, SONIA 2. This trial will last four years and will assess the long-term suitability of nitisinone for use in AKU patients. It will 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.

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 visit or contact our Clinical Trials Coordinator, Hana Ayoob. You can email her at or ring +44(0)1223 322897.

An Invitation To Join Our Upcoming Webinar!

From PatientsLikeMe & the AKU Society.
Don’t miss out on the chance to find out everything you need to know about our AKU community on PatientsLikeMe.
On Wednesday July 10th, we’re holding an exclusive webinar with PatientsLikeMe. It will allow AKU patients and caregivers the chance to get all their questions answered about our online community.Over 70 patients and caregivers have signed up to use the AKU community on PatientsLikeMe already. It allows you to take control of your disease, while connecting with others going through similar experiences.

Not only does it have an active AKU forum, but users are finding great use in the tools that PatientsLikeMe offers. Those signed up can track the progress of their AKU, create health profiles and evaluate their own treatments. Everything you do on PatientsLikeMe can be printed off for your doctor to see too.

The webinar will highlight the resources and tools available to you on the community. It will also show you how your input can help countless others with AKU learn from your experiences.

What the webinar will cover: 
• How you can connect with others who have AKU and share experiences in the AKU forum
• How to create a Doctor Visit Sheet to track your health between appointments
• What you can learn from the symptom and treatment reports
When: Wednesday, July 10th, 2013

Time: 4pm (GMT) Duration: 30 minutes

•  Liz Morgan, Head of Community, PatientsLikeMe
•  Jenni Thorburn, Online Communities Officer, AKU Society
Attendees can look forward to a guided tour of the AKU community from Liz, who will discuss how to use the site and what benefits it offers. Liz and Jenni will then hold a question and answer session, giving you the opportunity to find out all you need to know.

Register now and let us know you’re planning to attend. Do so by emailing Liz at, including any questions you may have for Liz and Jenni during the webinar.

After registering, you will receive a confirmation email containing information about joining the webinar. It will include a link on where to access the webinar on the day.

If you’re not already using the AKU community on PatientsLikeMe, why not sign up now and have a look around the community. 

We hope to see you all there!