DevelopAKUre Clinical Trials 

For up to date information about our clinical trials, please head to, our alkaptonuria clinical trials microsite.

The AKU Society and the Royal Liverpool University Hospital have launched a series of clinical trials to assess the use of the drug nitisinone in alkaptonuria (AKU) patients, partly funded through a grant from the European Commission.

Nitisinone is a drug currently licensed for use in another rare metabolic disease called hereditary tyrosinemia type 1 (HT-1), but it is yet to be approved for use in patients with AKU.

It's the world's first potential treatment for alkaptonuria.

It works by stopping the production of homogentisic acid (HGA), a toxic acid which causes the damage in AKU. Previous animal experiments and research studies have shown that nitisinone could be effective in treating AKU. Clinical research in the US showed that nitisinone reduced HGA levels by up to 95%. 

However, nitisinone isn't approved for use in alkaptonuria, meaning patients don't have widespread access to the drug. We don't know if treating patients with nitisinone will prevent complications and decrease disease. To find out and gain approval from the European Medicines Agency (EMA) for using this drug in AKU, we need to carry out a clinical trial. 

We are doing this to help people with AKU. If the trial is successful, we will be one step closer to getting nitisinone licensed for the treatment of AKU. And it could change our patients' lives.

We now have a website dedicated to information about the clinical trials. Find out more information by visiting We're also currently trying to raise the additional funds we desperately need for the clinical trials, primarily for patient and carer travel. Our patients will need to travel from all over the world to take part, perhaps with their carers or by specialist ambulance.

You can also find out more about the trials is by watching a recording of our latest clinical trials webinar below.

You can also watch the latest update video from Professor Ranganath here:

If you would like to receive further information, please get in touch. The best person to contact is our Patient Support Manager, Lesley Harrison. Contact her by emailing [email protected] or by phoning 01223 322897.



Find Your Way

Click Here to ( more...