Low-dose Ketamine for the Treatment of Rett Syndrome

David M. Katz, PhD, Case Western Reserve University & Dan Sessler, MD, Cleveland Clinic

$1,295,131 AWARDED

Case Western Reserve University and the Cleveland Clinic are recruiting for a Phase 2 clinical trial of low-dose ketamine for the treatment of Rett Syndrome. The study is being led by David Katz, Ph.D., Professor of Neurosciences and Psychiatry at Case Western and Daniel I. Sessler, M.D., Michael Cudahy Professor and Chair, Department of Outcomes Research at the Cleveland Clinic.

Studies undertaken by Dr. Katz have shown that low-dose ketamine can reverse deficits in brain activity in mouse models of Rett Syndrome in conjunction with significant improvements in neurological function, including breathing. Ketamine, a drug that has historically been used for sedation and anesthesia, has recently generated much enthusiasm for its ability to rapidly reverse major depression at low, sub-anesthetic, doses. This trial will determine the effect of multiple doses of ketamine on breathing abnormalities and overall clinical severity, as well as EEG abnormalities and repetitive behaviors.

Co-investigators include Daniel Sessler, M.D., Chairman, Department of Outcomes Research, Alparslan Turan, MD, Department of Anesthesiology, Kurt Ruetzler, MD, Department of Anesthesiology, Michelle Marks, DO, Department of Pediatrics, Prakash Kotagal, M.D., Department of Neurology, and Edward J. Mascha, Ph.D., Senior Biostatistician in the Department of Outcomes Research at the Cleveland Clinic.


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