35 Participants Needed

Antioxidant Cocktail for Rett Syndrome

RH
NP
KJ
LG
Overseen ByLisa Genore, HBSc
Age: < 65
Sex: Female
Trial Phase: Phase 2
Sponsor: Anagnostou, Evdokia, M.D.
Prior Safety DataThis treatment has passed at least one previous human trial

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

EA

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

My current medications for behavior have been stable.
Must have normal laboratory test results at Screening/Baseline, with clinically insignificant abnormal findings
Ability to complete assessments, fluency in English (parent/legal guardian; participant, if verbal)
See 3 more

Exclusion Criteria

Presence of another serious medical condition that might interfere with the study, confound interpretation of results, or endanger well-being
I am taking medications or supplements that contain Rett-T components.
Inability to tolerate venipuncture procedures for blood sampling
See 4 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

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

18 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Placebo
  • Rett-T
Trial Overview The study tests 'Rett-T,' an antioxidant cocktail, against a placebo to see if it helps with Rett syndrome's core motor issues. It also looks at safety and how the body responds to treatment by checking biological markers.
Participant Groups
2Treatment groups
Active Control
Placebo Group
Group I: Rett TActive Control1 Intervention
Rett T is a powder for oral suspension. Dosage is dependent on weight. For participants weighing \<30 kg, a 4 g dose (i.e., one 4 g sachet) is intended to be administered orally once a day after dissolving in approximately 125 mL of water. For participants weighing ≥30 kg, a 8 g dose (i.e., two 4 g sachets) is intended to be administered orally once a day after dissolving in approximately 250 mL of water.
Group II: PlaceboPlacebo Group1 Intervention
Placebo is a powder for oral suspension. Dosage is dependent on weight. For participants weighing \<30 kg, a 4 g dose (i.e., one 4 g sachet) is intended to be administered orally once a day after dissolving in approximately 125 mL of water. For participants weighing ≥30 kg, a 8 g dose (i.e., two 4 g sachets) is intended to be administered orally once a day after dissolving in approximately 250 mL of water.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Anagnostou, Evdokia, M.D.

Lead Sponsor

Trials
3
Recruited
180+

Unity Health Toronto

Collaborator

Trials
572
Recruited
470,000+

Holland Bloorview Kids Rehabilitation Hospital

Collaborator

Trials
69
Recruited
14,100+

Ontario Brain Institute

Collaborator

Trials
12
Recruited
3,000+

Findings from Research

In a double-blind crossover study involving 30 girls with Rett syndrome, mecasermin (rhIGF-1) did not show significant improvement in symptoms compared to placebo, and some measures indicated worsening of symptoms.
While the treatment was generally safe with mostly mild to moderate adverse events, serious adverse events were reported, and EEG parameters also showed deterioration, suggesting caution in its use for this condition.
Placebo-controlled crossover assessment of mecasermin for the treatment of Rett syndrome.O'Leary, HM., Kaufmann, WE., Barnes, KV., et al.[2019]

References

Placebo-controlled crossover assessment of mecasermin for the treatment of Rett syndrome. [2019]
Oxidative stress in Rett syndrome: natural history, genotype, and variants. [2021]
Effects of creatine supplementation in Rett syndrome: a randomized, placebo-controlled trial. [2013]
Functional outcomes in Rett syndrome. [2022]
F₄-neuroprostanes mediate neurological severity in Rett syndrome. [2015]
Oral Feeding of an Antioxidant Cocktail as a Therapeutic Strategy in a Mouse Model of Rett Syndrome: Merits and Limitations of Long-Term Treatment. [2022]
Blood oxidative stress and metallothionein expression in Rett syndrome: Probing for markers. [2018]
Effects of tocopherol and deprenyl on the progression of disability in early Parkinson's disease. [2022]
An open trial of high-dosage antioxidants in early Parkinson's disease. [2018]
10.United Statespubmed.ncbi.nlm.nih.gov
A pilot trial of high-dose alpha-tocopherol and ascorbate in early Parkinson's disease. [2019]
Epigallocatechin-3-Gallate Plus Omega-3 Restores the Mitochondrial Complex I and F0F1-ATP Synthase Activities in PBMCs of Young Children with Down Syndrome: A Pilot Study of Safety and Efficacy. [2021]
Neuroaspis PLP10™, a nutritional formula rich in omega-3 and omega-6 fatty acids with antioxidant vitamins including gamma-tocopherol in early Parkinson's disease: A randomized, double-blind, placebo-controlled trial. [2022]
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