SONIA 2, our second clinical trial, is currently ongoing and is fully recruited.
What is SONIA 2?
SONIA 2 stands for "Suitability of Nitisinone in Alkaptonuria 2" and is designed to test whether nitisinone slows 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 is essential to provide evidence for nitisinone slowing progression of AKU. Depending on the results of the study, and if nitisinone is shown to be effective, then the DevelopAKUre consortium could apply for a drug license as a treatment for AKU.
What happens during SONIA 2?
The trial is based at three clinical sites:
Liverpool (UK), Paris (France) and Piestany (Slovakia). Patients will make a total of six visits to a test centre, with each visit
lasting up to four days. They will also complete questionnaires between visits and there will be a follow-up phone call a month after
the last visit. SONIA 2 is expected to see its final patient in 2019.
Patients taking part in SONIA 2 are recruited from across Europe, and from Jordan. They have access to world experts in AKU at the three clinical sites and receive patient support from the AKU Society and ALCAP.
Why do we need SONIA 2?
There is some evidence that nitisinone works to treat AKU.However,
to be certain of its effectiveness, we need 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 will 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 should provide evidence on whether nitisinone has a positive impact on the amount of HGA in the body and if it can be used to help people with AKU.
For progress updates on the progress of SONIA 2 please visit the progress pages.
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