Vanderbilt University Medical Center to Lead Multisite Clinical Trial Comparing Three FDA-Approved Drugs for Rett Syndrome
Vanderbilt University Medical Center has been awarded a $13 million grant from the Department of Defense to spearhead a multisite clinical trial aimed at evaluating repurposed FDA-approved drugs as potential treatment options for patients suffering from Rett syndrome. Rett syndrome is a rare genetic neurodevelopmental disorder that affects brain development, with only 1 in 10,000 females at birth being impacted, and even fewer males.
The study, which falls under the umbrella of this trial, will compare three FDA-approved drugs – ketamine, vorinostat, and donepezil – against a common placebo. Participants will be randomly assigned to one of the four treatment arms and undergo a 16-week treatment cycle to assess safety, tolerability, efficacy, and biomarker response.
Jeffrey Neul, Annette Schaffer Eskind Professor and director of the Vanderbilt Kennedy Center, who serves as the principal investigator of the grant, explained the urgent need for new therapies to improve the lives of individuals with Rett syndrome. He highlighted the debilitating effects of the disorder, stating that 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. Neul emphasized that while symptom management, therapies, and a recently FDA-approved drug have shown some benefits, there remains a significant gap to be filled in the quest for more effective treatments.
The trial will take place across four trial sites, including Vanderbilt University Medical Center, Children’s Hospital of Philadelphia, Boston Children’s Hospital, and Children’s Hospital of Colorado, all designated as Rett Syndrome Centers of Excellence by the International Rett Syndrome Foundation (IRSF). Vanderbilt University Medical Center will be leading the coordination of the trial, leveraging their expertise in Rett syndrome research, as evidenced by their successful completion of a phase 3 trial that led to the first-ever FDA-approved drug for the condition.
Melissa Kennedy, CEO of IRSF, commended Vanderbilt’s dedication and contribution to Rett syndrome research and patient care. Kennedy expressed hope that the trial’s success would pave the way for more FDA-approved treatments, allowing individuals with Rett syndrome to do more and suffer less as soon as possible. She emphasized the importance of having multiple treatment options tailored to the complex nature of the disorder.
Furthermore, Neul highlighted the potential of the drugs being tested in the trial to benefit individuals with other neurological and mental health disorders as well. For instance, the study will contribute to building the safety profile of ketamine in the pediatric population, which is currently being investigated separately for the treatment of depression, post-traumatic stress disorder, and other conditions in adults.
The success of this clinical trial holds promise for individuals affected by Rett syndrome, as it may provide insights into new therapies and pave the way for the evaluation of potential treatments. With Rett syndrome being a challenging disorder, advancements arising from this trial can significantly impact the lives of affected individuals, offering hope for a brighter future.
In conclusion, Vanderbilt University Medical Center’s groundbreaking multisite clinical trial aims to assess the efficacy of repurposed FDA-approved drugs as treatment options for patients with Rett syndrome. By leveraging the expertise of multiple trial sites, including Vanderbilt University Medical Center and renowned children’s hospitals, this trial aims to improve the lives of individuals affected by Rett syndrome and potentially uncover benefits for other related disorders.
