Updated: Apr 3, 2019
November 2013 - Results of the Trial
The results from the Phase IIb trial of AFQ056 in adults with Fragile X Syndrome were released to researchers and participants in the study in November. This study was a four month trial which was looking to find out if AFQ056 was better than placebo for helping difficult behaviours in Fragile X Syndrome.
Disappointingly the main finding of the study was a negative one – AFQ056 was no more effective than placebo in helping people with Fragile X Syndrome. I know this will be disappointing news, not only for those people in the study, but also for others waiting to see what the results were going to be.
Although the drug did not work for adults, it is still possible that it may be helpful if people start taking it at younger ages - the brain is more flexible in younger people and may respond more easily to medications aimed at influencing its development. We should know if this is the case when the results of the trial of AFQ056 in adolescents (aged 12-17 years old) are available. These are expected in 2014.
The other purpose of the Phase IIb study was to look at whether AFQ056 was a safe medicine. No particular safety concerns were found in the study. The most common side effects were dizziness and difficulty getting to sleep which both occurred in about 1 in 5 people on the highest dose of the drug.
The Future for AFQ056
Once the results of the adolescent study are available, Novartis will make a decision about whether or not to continue investigating AFQ056.
There are some people who were in the Phase IIb trial who have since gone on to the longer term continuation study, where everyone gets the active medicine (i.e. there is no placebo). This continuation study will carry on for now so that if people want to they can keep taking the medicine (they can of course also leave the trial if they want to). It is likely that the continuation study will keep going until the results of the adolescent trial are available. At that point we expect a decision will be made about whether or not any of the trials will continue.
In the past Novartis have talked about doing a study in 5-11 year old children. At the moment we do not know whether this will still go ahead.
The Future for Research into Medical Treatments for Fragile X Syndrome
There are a number of other medicines that are currently being considered as potentially helpful for people with Fragile X Syndrome.
Some of them affect the brain in a similar way to AFQ056, some of them affect it differently. Although the results of the current trial are disappointing, the work towards finding medicines that help people with fragile X will continue.