Antioxidant Cocktail for Rett Syndrome
Trial Summary
Will I have to stop taking my current medications?
The trial requires that if you are already taking stable medications or supplements that affect behavior, you must continue on the same dose for at least one month before starting the trial and cannot change them during the study. However, if you are taking fluoxetine, you need to be on a stable dose for six weeks before the trial.
What data supports the effectiveness of the antioxidant cocktail treatment for Rett syndrome?
Research suggests that oxidative stress (damage caused by free radicals) plays a role in Rett syndrome, and omega-3 fatty acids, which are antioxidants, may help reduce this stress. This implies that an antioxidant cocktail could potentially be beneficial for managing symptoms of Rett syndrome.12345
Is the antioxidant cocktail safe for humans?
The antioxidant cocktail, which includes vitamin E, N-acetylcysteine, and alpha-lipoic acid, showed some benefits in mice with Rett syndrome, but long-term use may lead to issues like obesity and metabolic disturbances. This suggests that while it may help with some symptoms, careful monitoring is needed to avoid potential side effects.12467
How does the antioxidant cocktail treatment for Rett Syndrome differ from other treatments?
The antioxidant cocktail for Rett Syndrome is unique because it involves a combination of antioxidants, which are substances that can prevent or slow damage to cells caused by free radicals. This approach is different from standard treatments, as it focuses on reducing oxidative stress, a factor not typically targeted in Rett Syndrome therapies.89101112
What is the purpose of this trial?
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.
Research Team
Evdokia Anagnostou, MD
Principal Investigator
Holland Bloorview Kids Rehabilitation Hospital
Eligibility Criteria
This trial is for female outpatients aged 2-21 with Rett syndrome who can walk (with or without help). They should be on stable doses of any behavior-affecting meds, except fluoxetine which requires a longer period. Participants need normal lab results and must understand English to complete assessments.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either Rett-T or placebo for 8 weeks, followed by a 2-week washout period, and then switch to the other treatment for another 8 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Placebo
- Rett-T
Find a Clinic Near You
Who Is Running the Clinical Trial?
Anagnostou, Evdokia, M.D.
Lead Sponsor
Unity Health Toronto
Collaborator
Holland Bloorview Kids Rehabilitation Hospital
Collaborator
Ontario Brain Institute
Collaborator