As many are anticipating an update on the long-awaited Clinical Trial in Canada, we felt that October was the best time to provide everyone an update on the Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents with Rett Syndrome
As we are aware, 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 behaviour 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.
This study is an 18-week pilot, single site, randomized, double blind, placebo-controlled cross-over trial of Rett-T vs. placebo. Periods I and II of the randomized study are 8 weeks long with a 2-week washout period. The Holland Bloorview Kids Rehabilitation Hospital (Toronto) will be starting to recruit in the beginning of 2020. The manufacturing company has confirmed the production of the Antioxidant Cocktail in advance of the recruiting process.
For the full details of the Clinical Trial, please visit: https://clinicaltrials.gov/ct2/show/NCT04041713…