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

Launched by ANAGNOSTOU, EVDOKIA, M.D. · Jul 30, 2019

Keywords

ClinConnect Summary

This clinical trial is looking at a treatment called Rett-T, which is a mix of antioxidants, to see if it can help improve movement issues in children and teenagers with Rett syndrome. The study will also check for safety and look at biological markers to understand how well the treatment is working. The trial is currently not recruiting participants, but when it does, it will focus on girls aged 2 to 21 who have been diagnosed with Rett syndrome and can walk, even if they need some help.

To be part of the study, participants must be stable in any existing treatments for at least a month before joining, and they should not have any serious health problems that could affect their safety during the trial. Participants will undergo assessments and provide consent from their parents or guardians. This study is an important step in exploring new treatment options for Rett syndrome, which could potentially lead to better care for those affected by this condition.

Gender

FEMALE

Eligibility criteria

• 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.