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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

35 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-10-01

Study Completion Date

2026-11-30

Brief Summary

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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.

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Detailed Description

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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

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Rett Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

This 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.

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Study Groups

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Rett T

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 Type ACTIVE_COMPARATOR

Rett-T

Intervention Type DRUG

antioxidant cocktail

Placebo

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.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Placebo

Interventions

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Rett-T

antioxidant cocktail

Intervention Type DRUG

Placebo

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

1. Female outpatients 2-21 years of age inclusive.
2. Diagnosis of Rett syndrome.
3. At least partially ambulatory (may need assistive device to take a step).
4. If already receiving stable interventions must meet the following criteria:

* If already receiving stable concomitant medications or nutraceuticals affecting behaviour, must be on a stable dose during the preceding 1 month prior to Screening (with the exception of fluoxetine, where a period of 6 weeks is needed), and will not electively initiate new or modify ongoing medications for study duration.
5. Have normal laboratory test results at Screening/Baseline. If abnormal, the finding(s) must be deemed clinically insignificant by the Investigator.
6. Ability to complete assessments, fluency in English (parent/legal guardian; participant, if verbal).
7. Ability to obtain assent (if developmentally appropriate), as well as written informed consent from their parent(s)/legal guardian(s).

Exclusion Criteria

1. Pregnant females; sexually active females on inadequate birth control (extremely unlikely in this population).
2. Have another serious medical condition that, based on Investigator judgment, might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. Have evidence or history of malignancy or any significant hematological, endocrine, cardiovascular (including any rhythm disorder), respiratory, renal, hepatic, or gastrointestinal disease, not including mild common pediatric diseases in these areas that are stable (e.g. mild asthma, constipation, etc.).
3. Have hypersensitivity to any components of Rett-T.
4. Have one or more of the following: HIV, HBV, HCV, hemophilia (bleeding problems, recent nose and brain injuries), drug abuse, immunity disorder, major depressive episode or psychosis.
5. Unable to tolerate venipuncture procedures for blood sampling.
6. Receiving concomitant medications/nutraceuticals that include any of the components of Rett-T.
7. Actively enrolled in another intervention study.

Minimum Eligible Age

2 Years

Maximum Eligible Age

21 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No