Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Patients

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.

Inclusion Criteria:Females age 18-45 (inclusive)Diagnosis of classic/typical Rett syndrome with a documented mutation of the MeCP2 geneSeverity 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 enrollmentAble 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 historyExcluded concomitant medicationsCurrent clinically significant (as determined by the investigator). cardiovascular, endocrine, hepatic, renal, or respiratory diseaseGastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medicationHistory of, or current cerebrovascular disease or brain traumaHistory of, or current, malignancyClinically significant abnormalities in safety laboratory tests, vital signs, or ECG, as measured at screening or baselineAny condition which in the investigator's opinion would affect the ability of the subject to participate in the studyAllergy to VYNT-0126 or any ingredients of the liquid formulation