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

Study Overview
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Brief Summary
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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.

Official Title
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Phase 2, Double-Blind, Randomized, Placebo-Controlled Clinical Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Female Patients

Conditions
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Rett Syndrome

Intervention / Treatment
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* Drug: VYNT-0126
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 45 Years

Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br> | Drug: Placebo<br>* Liquid for oral administration once daily<br>|
| Experimental: 5 mg VYNT-0126<br> | Drug: VYNT-0126<br>* Liquid for oral administration once daily<br>|
| Experimental: 10 mg VYNT-0126<br> | Drug: VYNT-0126<br>* Liquid for oral administration once daily<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | 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 | 

Secondary Outcome Measures

| Outcome Measure | 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). | 
| 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). |