Registering your child or adult with Rett Syndrome on the UK Rett Syndrome Patient Registry at Reverse Rett is the best way to access UK clinical trials.
The Registry is the first place we look for patients for upcoming trials. When a trial is imminently about to start recruiting, the families of all registered patients who meet initial criteria (e.g. age group and mutation positive) are contacted so that they can confirm their interest.
It’s also critical that people whose children do not meet the criteria for a particular trial, register and formally express interest in the trial. Having information about how many patients there are in other age groups for example, will enable us to demonstrate that there is an unmet need, which could lead to amendments in trial protocol which could enable participation.
There are obviously some elements of trial protocol where an amendment is not possible. Clinical trial protocol is designed to get the best possible results from a specified group in order to demonstrate that a new drug is safe and that it works.
For those who for whatever reason, have children who do not meet basic criteria for current UK trials, please take heart. New drugs for Rett being licensed depends highly on the success of clinical trials. We need them to be as successful as possible in a relatively small pool of patients, so that the wider patient group can access them. Sadly, this will mean that not everyone can take part at the clinical trial level.
Non-eligibility for a trial, however, does not necessarily mean that patients will not be able to access a specific drug if, as and when it is licensed.
At Reverse Rett we are always working to facilitate access to treatments for UK patients with Rett Syndrome of all ages, genders and backgrounds.
That’s why it is critical that we have up to date information about the patients living in the UK today, where they are and who we can contact to let them know as clinical trials, treatments and ultimately cures emerge.