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

NCT04041713 | Not Yet Recruiting Phase 2

• 1 other identifier: RETT-08-2018
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Rett Syndrome

Keywords

Rett Syndrome; Rett; RTT

Outcome Measures

Primary Outcomes (1)

• Rett Syndrome Natural History Motor Behavior Assessment (MBA)

  To examine the effect of Rett-T vs. placebo on measures of motor function. The Rett Syndrome MBA consists of 37 items grouped into three subscales (Behavioral/Social Assessment, Orofacial Respiratory Assessment, and Motor Assessment/Physical Signs). Items are captured on a 0-4 point Likert scale. Total and subscale scores are calculated (subscales are summed for a total score). The scale range for Total score is 0-136; for Behavioral/Social subscale, 0-60; for Orofacial/Respiratory subscale, 0-28; for Motor Assesment/Physical Signs, 0-48. For both total and subscale scores, higher values represent a worse outcome.

  18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim

Secondary Outcomes (7)

• Rett Syndrome Gross Motor Scale (RSGMS)

  18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim

• Safety Monitoring Uniform Report Form (SMURF)

  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)

  18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim

• The Top 3 Causes for Concern

  18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim

• The Rett Syndrome Behaviour Questionnaire (RSBQ)

  18 weeks total: 8 weeks on Rett-T, 8 weeks on placebo, 2 week washout in interim

Study Arms (2)

Rett T

ACTIVE COMPARATOR

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.

Drug: Rett-T

Placebo

PLACEBO COMPARATOR

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.

Other: Placebo

Interventions

Rett-T DRUG

antioxidant cocktail

Rett T

Placebo OTHER

Placebo

Placebo

Eligibility Criteria

• Age: 2 Years - 21 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Female outpatients 2-21 years of age inclusive.
• Diagnosis of Rett syndrome.
• At least partially ambulatory (may need assistive device to take a step).
• 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.
• Have normal laboratory test results at Screening/Baseline. If abnormal, the finding(s) must be deemed clinically insignificant by the Investigator.
• Ability to complete assessments, fluency in English (parent/legal guardian; participant, if verbal).
• Ability to obtain assent (if developmentally appropriate), as well as written informed consent from their parent(s)/legal guardian(s).

You may not qualify if:

• Pregnant females; sexually active females on inadequate birth control (extremely unlikely in this population).
• 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.).
• Have hypersensitivity to any components of Rett-T.
• 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.
• Unable to tolerate venipuncture procedures for blood sampling.
• Receiving concomitant medications/nutraceuticals that include any of the components of Rett-T.
• Actively enrolled in another intervention study.

Sponsors & Collaborators

• Holland Bloorview Kids Rehabilitation Hospital: lead
• Ontario Brain Institute: collaborator
• Unity Health Toronto: collaborator

Study Sites (1)

Holland Bloorview Kids Rehabilitation Hospital

Toronto, Ontario, M4G 1R8, Canada

Location

MeSH Terms

Conditions

Rett Syndrome

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System

Study Officials

• Evdokia Anagnostou, MD

  Holland Bloorview Kids Rehabilitation Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Karly Janisse, BSc

CONTACT

416 425 6220; kjanisse@hollandbloorview.ca

Lisa Genore, HBSc

CONTACT

416 425 6220; lgenore@hollandbloorview.ca

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: June 27, 2019
• First Posted: August 1, 2019
• Study Start: October 1, 2025
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): November 30, 2026
• Last Updated: July 16, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)