This is an exploratory, Phase 2, multicenter, double-blind, parallel-group, placebo-controlled study to assess the safety, tolerability, and efficacy of oral treatment with VYNT-0126 in female subjects 18-45 years of age with Rett syndrome.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
48
Adverse Events
Incidence of adverse events (AEs), including serious adverse events (SAEs), will be compared across the two VYNT-0126 doses and placebo. SAEs and AEs will be examined throughout the study.
Time frame: Through study completion, approximately 14 weeks
Change from Baseline in the Rett Syndrome Behavioral Questionnaire (RSBQ)
Time frame: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
Change from Baseline in the 24-Item Motor-Behavioral Assessment (MBA)
Time frame: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
Change from Baseline in Clinical Global Impression of Severity (CGI-S)
Time frame: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
Clinical Global Impression of Improvement (CGI-I)
Time frame: Obtained at the end of dose titration and end of treatment (approximately 14 weeks).
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