There have never been more opportunities to help develop new drugs for Rett than there are today. Because of all the promising new research, as many people with Rett Syndrome as possible will be needed to participate in trials. Under-enrolment in trials is one of the biggest challenges for researchers and trial sponsors, and it can slow the development of a potential treatment. There are only an estimated 2500 individuals with Rett Syndrome in the UK. That’s why it’s critical that those who are interested in participating in trials register on the UK Rett Syndrome Patient Registry.
Participating in a clinical trial can be a very satisfying and worthwhile experience. People choose to participate in clinical trials for a number of different reasons. Some possible benefits include: – Gaining access to new treatments before they are more widely available – Receiving a treatment that works for your child or adult with Rett – Taking a more active role in managing your child or adult’s health care – Helping advance our knowledge of Rett Syndrome.
Although there are many benefits to participating in a clinical trial, it is important to remember there also are risks. Possible risks include: – Side effects of the medications or treatments being studied – Unwanted events during the trial that may or may not be related to the study drug – Failure of a treatment to work The research team will continuously monitor your or your child’s health and safety throughout the trial, whether you are receiving the drug being studied or a placebo. Remember: You can leave a study at any time. Those who are monitoring the trial for safety — such as the principal investigator can pull your child from a study if they believe their health is declining. People with Rett Syndrome and their families who take part in clinical trials are pioneers. They are helping to beat a path to better treatments for Rett for all of us. Register your child or adult with Rett today so that you can stay informed as and when opportunities to participate become available.
It’s a big decision to take part in a clinical trial for any reason, particularly when the trial centre is hundreds of miles from your home. Professor Santosh and the team have understood the logistical impact of every visit we make to King’s, and Emily’s well-being has been paramount at every step of the journey, which is very reassuring. The practical assistance from the Reverse Rett team to make sure accommodation is booked and appropriate to our needs, takes much of the stress out of the process. We knew there were no guarantees when we started the trial. Emily has been committed to this trial from the start, despite the travel, inconvenience and occasional discomfort caused by taking part. I think this shows just how much Emily values a treatment for her breath-holding and hyperventilation. It’s exciting to be part of a breakthrough in treating one of the distressing symptoms of Rett Syndrome.
Mother to Emily, age 20
If you feel helpless, or powerless or hopeless in the face of a Rett diagnosis, please add the person you care for to our UK Rett Syndrome Patient Registry. Signing up means we can contact you directly about clinical trials, the latest research and enables us to make a better case for improved clinical provision and better treatments to become available in the UK.
Hospital visits can be an ordeal for children and adults with Rett and their families. From the basics like quickly informing treating clinicians how best to take blood, to more complex information like flagging up potential drug interactions, we hope the Reverse Rett hospital passport will help.
Reverse Rett are able to supply you with information which can help you keep your child or adult with Rett safe and healthy until better treatments and our cure become available.