Rett Syndrome
Conditions
Brief summary
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.
Interventions
DRUGVYNT-0126
Liquid for oral administration once daily
DRUGPlacebo
Liquid for oral administration once daily
Sponsors
Vyant Bio
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
FEMALE
Age
18 Years to 45 Years
Healthy volunteers
No
Inclusion criteria
* Females age 18-45 (inclusive) * Diagnosis of classic/typical Rett syndrome with a documented mutation of the MeCP2 gene * Severity rating of between 10 and 36 (Rett Syndrome Natural History/Clinical Severity Scale) * Concomitant medications (including approved medications for treatment of Rett syndrome) must be stable for \>4 weeks prior to enrollment * Able to receive liquid study drug orally or via gastrostomy tube (G-tube)
Exclusion criteria
* Actively undergoing neurological regression; * Abnormal QT interval, prolongation or significant cardiovascular history * Excluded concomitant medications * Current clinically significant (as determined by the investigator). cardiovascular, endocrine, hepatic, renal, or respiratory disease * Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication * History of, or current cerebrovascular disease or brain trauma * History of, or current, malignancy * Clinically significant abnormalities in safety laboratory tests, vital signs, or ECG, as measured at screening or baseline * Any condition which in the investigator's opinion would affect the ability of the subject to participate in the study * Allergy to VYNT-0126 or any ingredients of the liquid formulation
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse Events | Through study completion, approximately 14 weeks | 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. |
Secondary
| Measure | 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). |
| 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). |
Other
| Measure | Time frame | Description |
|---|---|---|
| Quantitative Electroencephalograms (EEGs) | Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks). | Quantitative EEGs will be explored as potential biomarkers of intervention effects on brain function and clinical severity. |
| Electrophysiological Evoked Potentials | Obtained at baseline, 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. |
Outcome results
None listed