SONIA 1

 
SONIA 1 was completed in June 2014. The main scientific paper from the study was published in December 2014 in the Annals of Rheumatic Diseases. The paper is available online but requires a subscription to view.
 
What was SONIA 1?
 
SONIA 1 stood for “Suitability of Nitisinone in Alkaptonuria 1” and was designed to find out what would be the correct dose of nitisinone to reduce levels of homogentistic acid (HGA) in the body. 

40 patients were recruited for the trial and were split up into five groups of eight. Each group received varying doses of nitisinone, or non-treatment. The levels of HGA were monitored and the results of each group compared.

SONIA 1 began in May 2013 and lasted for four weeks. There were two centres for the trial: the Royal Liverpool University Hospital in the UK, and the National Institute of Rheumatic Disease in Piešťany, Slovakia. Patients were required to make three trips to their allocated centre: one at the start of the four-week trial, one after two weeks, and another at the end.
 

 
Why did we need SONIA 1?
 
Clinical research in the US suggests that nitisinone is capable of reducing levels of homogentisic acid by up to 95%.
 
SONIA 1 was designed to identify the most appropriate dose of nitisinone to be used over a patient's lifetime, as well as the most effective dose in reducing levels of HGA.
 
The resulting dosage is now being used as the investigatory treatment for the second trial (SONIA 2). 
 
What were the results?
 
Ultimately the importance of SONIA 1 was to show evidence that nitisinone reduces HGA, and to choose the best dose to take forward into SONIA 2 (our current clinical trial).

SONIA 1 concluded that treatment with nitisinone in AKU patients does reduce HGA, and the reduction is dependent on dose. This means a larger dose of nitisinone results in a greater reduction of HGA. 

The trial helped us to understand that 10 mg of nitisinone is the best dose to test the drug’s effectiveness in AKU patients. This is now the dose we are using for SONIA 2. 

Prof Jim Gallagher from the University of Liverpool said “This is an outstanding example of impact in translational research, involving collaboration between clinical and basic scientists, patient organisations and pharma. Initially we demonstrated that nitisinone was completely effective and safe in AKU mice. We have now shown that it is an effective HGA-lowering therapy in AKU patients.”

While the study didn’t look especially at safety, there were no safety concerns at all during SONIA 1. 
 

 
 

Welcome To DevelopAKUre


DevelopAKUre is a series of major international clinical trials, run by a consortium of 12 European partners. It aims to study a potential new drug, called nitisinone, and assess its potential effectiveness in treating the rare disease, alkaptonuria (AKU).

DevelopAKUre is co-funded by a grant from the European Commission. This website is run by a UK patient group, the AKU Society. Learn more about AKU on the AKU Society's What is AKU page.

 

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