Department of Defense Grant to Power Multisite Trial of Rett Syndrome Therapy


Initiative seeks to find new therapies to improve the lives of patients with Rett syndrome.

Image credit: Vitalii Vodolazskyi |

Image credit: Vitalii Vodolazskyi |

Vanderbilt University Medical Center (VUMC) has received a $13 million grant from the Department of Defense to lead a multisite clinical trial that will evaluate repurposed FDA-approved drugs as treatment options for patients with Rett syndrome.

According to VUMC, Rett syndrome affects 1 in 10,000 females at birth and is even more rare in males. It is a genetic neurodevelopmental disorder that affects brain development.

“It robs affected individuals of the ability to use their hands or speak and causes problems with mobility, as well as a number of other issues,” said the grant’s principal investigator Jeffrey Neul, Annette Schaffer Eskind Professor, director of the Vanderbilt Kennedy Center, in the press release. “People with Rett syndrome require lifelong care for activities of daily living. Although symptom management, therapies and a newly FDA-approved drug can help, there remains a great need to find new therapies to improve the lives of people with Rett syndrome.”

The umbrella trial will compare ketamine, vorinostat, and donepezil to a common placebo. Participants will be randomly assigned equally across the four treatment paths for 16 weeks to assess safety and efficacy among other biomarker responses.

“The study will evaluate FDA-approved drugs that have preclinical evidence supporting potential clinical benefit,” Neul continued in the press release. “If any of these drugs show benefit in this trial, further clinical evaluation and ultimately use treating people with Rett syndrome could progress rapidly. Additionally, successful establishment of this clinical trial platform will allow accelerated evaluation of other potential therapies in Rett syndrome.”

Participants will be recruited from four trial sites, including VUMC and all International Rett Syndrome Foundation (IRSF)-designated Rett Syndrome Centers of Excellence. The trial will be led and coordinated by VUMC, which has led a number of trials in Rett syndrome including a recent Phase III trial that led to the first FDA-approved drug for this condition.

“Vanderbilt has a long history of providing best-in-class care to Rett syndrome patients as one of only 18 Rett clinics across the country that IRSF designated as a Center of Excellence,” said Melissa Kennedy, chief executive officer of IRSF in the press release. “Dr. Neul and the Vanderbilt team have proven themselves as research leaders as well, conducting clinical research and coordinating trials that have not only expanded our understanding of the fundamental science behind Rett syndrome but also played a pivotal role in delivering the first-ever FDA-approved treatment.”

Neul also noted that the drugs in this trial may uncover benefits for other neurological and mental health disorders, according to the press release. This includes studying the safety of ketamine in pediatric patients, which is also being studied separately for the treatments of depression, post-traumatic stress disorder, and other conditions in adults.


1. Multisite clinical trial will compare three FDA-approved drugs for Rett syndrome treatment. (2023, January 16). EurekAlert.

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