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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator |
| |
| 5 mg VYNT-0126 | Experimental |
| |
| 10 mg VYNT-0126 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VYNT-0126 | Drug | Liquid for oral administration once daily |
| |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Through study completion, approximately 14 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in the Rett Syndrome Behavioral Questionnaire (RSBQ) | 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) | Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks). |
| Measure | Description | Time Frame |
|---|---|---|
| Quantitative Electroencephalograms (EEGs) | Quantitative EEGs will be explored as potential biomarkers of intervention effects on brain function and clinical severity. | Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks). |
| Electrophysiological Evoked Potentials |
Inclusion Criteria:
Exclusion Criteria:
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| ID | Term |
|---|---|
| D015518 | Rett Syndrome |
| ID | Term |
|---|---|
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
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| Placebo |
| Drug |
Liquid for oral administration once daily |
|
| Change from Baseline in Clinical Global Impression of Severity (CGI-S) | Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks). |
| Clinical Global Impression of Improvement (CGI-I) | Obtained at the end of dose titration and end of treatment (approximately 14 weeks). |
Evoked potential following auditory and visual stimuli will be explored as potential biomarkers of intervention effects on brain function and clinical severity. |
| Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks). |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |