NCT04041713

A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 35 (estimated)
• Sponsor: Holland Bloorview Kids Rehabilitation Hospital · Academic / Other
• Sex: Female
• Age: 2 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Rett Syndrome

Interventions

Type	Name	Description
DRUG	Rett-T	antioxidant cocktail
OTHER	Placebo	Placebo

Timeline

• Start date: 2025-10-01
• Primary completion: 2026-11-30
• Completion: 2026-11-30
• First posted: 2019-08-01
• Last updated: 2025-07-16

Locations

1 site across 1 country: Canada