1. Why should I consider participating in the ketamine study?

Independent labs have shown positive effects of ketamine treatment in different Rett mouse models, in both females and males, including improved brain cell function and activity, improved sensorimotor function (body movements in response to sensory input), less apnea (breath holds) and longer life-span. With the exception of gene therapy, Ketamine treatment extends life-span in animal models longer than any other drug currently in development for Rett.

Ketamine affects many aspects of brain function, although it does not affect the mutation (change in DNA) in the MECP2 gene that causes Rett Syndrome. In animal models of Rett Syndrome, some of the positive changes in the brain after ketamine treatment seem to last for at least some time after the drug has left the body.

One person with Rett syndrome received low-dose ketamine to treat seizures and showed improvement in other Rett symptoms as well. Participating in this study will help us answer the question if ketamine can be used to treat symptoms of Rett Syndrome generally, or if her response was unique.

Ketamine is an FDA-approved medication for anesthesia (insensitivity to pain) and is used in surgery and other medical procedures for inducing sleep and to reduce pain. Because ketamine has been used to treat patients for over 50 years, there is a large amount of information available regarding safety and possible side effects in adults and children who have received ketamine.

Over the past 20 years low dose ketamine has been studied in disorders affecting brain function such as depression, anxiety and Obsessive Compulsive Disorder (OCD), as well as in pain management, with positive effects.

Participating in this study will not prevent you from participating in other studies afterward. You will need to wait about 1 week after the final phone call in this study before you can join another study. Because this study is short and lasts about 2 months, you will have plenty of time to join other studies.