The Programme

The DevelopAKUre programme is taking place at a select few clinics across Europe and aims to assess the effectiveness of the drug nitisinone in treating alkaptonuria (AKU).

Nitisinone is currently licensed to treat hereditary tyrosinemia type 1 (HT-1), another rare disease. However, research has indicated the drug could also potentially be the first effective treatment for AKU. Laboratory experiments have shown nitisinone could stop the progression of the disease (6), while clinical research in the US resulted in nitisinone reducing levels of homogentisic acid (HGA) by up to 95% (7).

The DevelopAKUre programme consists of three trials:

1. Phase II dose-response trial (SONIA 1: Suitability Of Nitisinone In Alkaptonuria 1)
• To determine the useful dose-range in the treatment of AKU

2. Phase III clinical efficacy trial (SONIA 2: Suitability Of Nitisinone In Alkaptonuria 2)
• Evaluation of the long-term efficacy and safety of nitisinone for treatment of AKU

3. Cross-sectional trial (SOFIA: Subclinical Ochronosis Features In Alkaptonuria)
• To determine the age at which treatment should begin

The complete programme will last around five-and-a-half years and will require a maximum of 212 participating individuals. We are currently seeking AKU patients from across Europe to participate in our third study, SOFIA. This will begin this summer.

If you are interested in participating, or would like to find out more information about the programme, please do so by registering your interest in the form at the bottom of the page. Our Patient Support Manager, Lesley Harrison, will be in touch.

If you’re unsure about participating, an appointment with your regular doctor may help you to decide. In addition, the trial centre staff will be pleased to provide you with more information about the programme and answer any of your questions.