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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT04041713
• Date of registration: 27/06/2019
• Prospective Registration: Yes
• Primary sponsor: Anagnostou, Evdokia, M.D.
• Public title: A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome
• Scientific title: A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome
• Date of first enrolment: September 1, 2019
• Target sample size: 35
• Recruitment status: Not yet recruiting
• URL: https://clinicaltrials.gov/show/NCT04041713
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Canada

Contacts

• Name: Evdokia Anagnostou, MD
• Affiliation: Holland Bloorview Kids Rehabilitation Hospital

• Name: Rianne Hastie Adams, MSW

• Name: Rianne Hastie Adams, MSW

Key inclusion & exclusion 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.



Age minimum: 2 Years
Age maximum: 21 Years
Gender: Female

Health Condition(s) or Problem(s) studied

Rett Syndrome

Intervention(s)

Other: Placebo

Drug: Rett-T

Primary Outcome(s)

Rett Syndrome Natural History Motor Behavior Assessment (MBA) [Time Frame: 18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim]

Secondary Outcome(s)

The Top 3 Causes for Concern [Time Frame: 18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim]

Rett Syndrome Gross Motor Scale (RSGMS) [Time Frame: 18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim]

Children's Sleep Habits Questionnaire (CSHQ) [Time Frame: 18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim]

Clinical Global Impressions - Improvement Scale - Global (CGI-I) [Time Frame: 18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim]

Safety Monitoring Uniform Report Form (SMURF) [Time Frame: 18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim]

Anxiety, Depression, and Mood Scale (ADAMS) [Time Frame: 18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim]

The Rett Syndrome Behaviour Questionnaire (RSBQ) [Time Frame: 18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim]

Secondary ID(s)

RETT-08-2018

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Holland Bloorview Kids Rehabilitation Hospital

St. Michael's Hospital, Toronto

Ontario Brain Institute