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

Sponsor
Holland Bloorview Kids Rehabilitation Hospital
Study ID
NCT04041713
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
FEMALE
Age
2 Years - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • Rett-T — DRUG
    antioxidant cocktail
  • Placebo — OTHER
    Placebo

Study Details

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.

Key Dates

First listed
Aug 1, 2019
Start date
Aug 24, 2026
Status verified
Jul 2026
Primary completion
Dec 30, 2027
Completion
Dec 30, 2027

Study Design

Enrollment
35 participants (estimated)
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT

Arms

  • Active Comparator: Rett T
    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.
  • Placebo Comparator: Placebo
    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.

Primary Outcome Measure

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 ]

Central Contacts

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