SONIA 2, our second clinical trial, finished in January 2019
What is SONIA 2?
SONIA 2 stood for "Suitability of Nitisinone in Alkaptonuria 2" and was designed to test whether nitisinone slowed down the damage that AKU does to the body and if it is safe to use for AKU patients.
SONIA 2 enrolled 138 patients who have been randomly divided into two equal groups. 69 receive nitisinone and the other 69 receive no treatment. Comparing the two groups was essential in providing evidence for nitisinone slowing progression of AKU.
SONIA 2 has now been analysed and very positive trends have been highlighted. Due to this, SOBI (Swedish Orphan Biovitrum) announced that they are going to apply to the European Medicines Agency (EMA) for market authorisation for the drug to be used in treating AKU. If successful, this could lead to nitisinone being licenced and available to all AKU patients in Europe.
What happened during SONIA 2?
The trial was based at three clinical sites:
Liverpool (UK), Paris (France) and Piestany (Slovakia). Patients made a total of six visits to a test centre, with each visit
lasting up to four days. They also completed questionnaires between visits and a follow-up phone call a month after
the last visit.
Patients taking part in SONIA 2 were recruited from across Europe, and from Jordan. They have access to world leading experts in AKU at the three clinical sites and receive patient support from the AKU Society and ALCAP.
Why did we need SONIA 2?
There was some evidence that nitisinone works to treat AKU. However,
to be certain of its effectiveness, we needed to prove that the drug is able to lower HGA in AKU patients and show that it is safe for
use as a treatment in AKU.
Our previous trial, SONIA 1, showed that if we used the correct dose of nitisinone, HGA levels were reduced dramatically. SONIA 2 was planned to underline the impact the drug has on levels of HGA in the body over four years and compare it with AKU patients who are not taking nitisinone. This provided the evidence that nitisinone had a positive impact on the amount of HGA in the body and if it can be used to help people with AKU.
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.
In today’s blog Chief investigator Prof. Ranganath reinforces the need for SONIA 2 pat.. ...more