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

Brief Title

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

Official Title

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

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.
    

Detailed Description

      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.
    

Study Phase

Phase 2

Study Type

Interventional

Primary Outcome

Rett Syndrome Natural History Motor Behavior Assessment (MBA)

Secondary Outcome

 Rett Syndrome Gross Motor Scale (RSGMS)

Condition

Rett Syndrome

Intervention

Rett-T

Study Arms / Comparison Groups

 Rett T

Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Drug

Estimated Enrollment

35

Start Date

September 1, 2019

Completion Date

January 31, 2021

Primary Completion Date

January 31, 2021

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.
      

Gender

Female

Ages

2 Years - 21 Years

Accepts Healthy Volunteers

No

Contacts

Evdokia Anagnostou, MD, 416 425 6220, [email protected]

Location Countries

Canada

Location Countries

Canada

NCT ID

NCT04041713

Organization ID

RETT-08-2018

Responsible Party

Sponsor

Study Sponsor

Anagnostou, Evdokia, M.D.

Collaborators

 Ontario Brain Institute

Study Sponsor

Evdokia Anagnostou, MD, Principal Investigator, Holland Bloorview Kids Rehabilitation Hospital

Verification Date

August 2019