Rett Syndrome Clinical Trial
Official title:
A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome
This study will examine the potential efficacy and safety of Rett-T for core motor deficits of Rett syndrome, and will explore biological markers of safety and treatment response.
There are currently no available medicines shown to be effective for Rett syndrome. Numerous
studies implicate mitochondrial dysfunction and oxidative stresses in the pathophysiology of
Rett syndrome. Mitochondrial dysfunction has been reported in Rett patients, Rett mouse
models and MECP2-deficient cells.
Collaborators have tested a combination of specific antioxidants known to enhance
mitochondrial function in a cell and mouse model of Rett syndrome. The formulation normalized
mitochondrial membrane potential in MECP2 neurons, and MECP2-deficient mice displayed
improved exploratory, locomotor and social behavior compared to MECP2-deficient mice. These
results support testing anti-oxidative strategies for benefit in individuals with Rett
syndrome.In this study, the formulation has been adjusted and optimized based on current
guidelines for human use, with the goal of translating a potential new treatment from the
animal model to use in humans. Results of this study could lead to the first approved
medication treatment for the disorder.
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