Antioxidant Cocktail vs. Placebo for Rett Syndrome in Children and Teens

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 2 interventional study will evaluate the safety and potential benefit of Rett-T compared with placebo in female outpatients aged 2–21 with Rett syndrome. The investigational treatment is designed to support mitochondrial function and reduce oxidative stress, based on preclinical data suggesting improvements in motor and behavioral aspects. The trial will monitor safety through lab tests and adverse events, and explore biological markers related to safety and treatment response. Eligible participants should be ambulatory to some degree and able to complete assessments, with assent and parental consent required.

Why it matters

Who can join: the full rules

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.

Where it's running

• Holland Bloorview Kids Rehabilitation Hospital
  Toronto, Ontario, Canada Recruiting

Study contacts

• Principal investigator: Evdokia Anagnostou, MD — Holland Bloorview Kids Rehabilitation Hospital
• Study coordinator: Faiza Khawaja
• Email: fkhawaja@hollandbloorview.ca
• Phone: 416 425 6220

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Auditory EEG and behavioral testing for people with Rett syndrome

  This project will record noninvasive EEG while people with Rett syndrome and matched controls listen to simple sounds to see how their brains process sound.

  Recruiting University of Rochester · Rochester, New York · NCT07569445

• Fenfluramine HCl for Rett Syndrome in a Phase 3 study

  This study tests whether fenfluramine hydrochloride helps people with Rett syndrome when compared to a placebo.

  Recruiting at 40 sites Phase 3 UCB Pharma · Little Rock, Arkansas and 39 other locations · NCT07503444

• Daily oral vorinostat for girls and young women with Rett syndrome

  This trial will try daily oral vorinostat at two escalating dose levels, using a four-week placebo baseline and an N-of-1 design so each participant serves as her own control, to see if the drug is safe and shows clinical or molecular benefit in girls and young women with typical Rett syndrome.

  Recruiting Phase 1 Unravel Biosciences, Inc. · Medellín · NCT07150013

• Efficacy of Bionetide for treating Rett syndrome in females

  This study is testing if an oral medication called Bionetide can help improve behavior and communication in girls and young women aged 5 to 20 with Rett syndrome.

  Recruiting at 18 sites Phase 3 Biomed Industries, Inc. · Phoenix, Arizona and 17 other locations · NCT06840496

• Assessing cognitive function in Rett syndrome patients receiving trofinetide treatment

  This study is testing how well eye-tracking tasks can measure cognitive abilities in people with Rett syndrome who are starting treatment with trofinetide.

  Recruiting University of Minnesota · Minneapolis, Minnesota · NCT06705816

• Identifying biomarkers in Rett Syndrome

  This study is trying to find specific brain signals in girls with Rett Syndrome to see how they differ from girls without the condition and how these signals relate to the severity of their symptoms over time.

  Recruiting at 6 sites Children's Hospital of Philadelphia · Los Angeles, California and 5 other locations · NCT05932589

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.