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

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

Study Overview

• Status: Not yet recruiting
• Conditions: Rett Syndrome
• Intervention / Treatment: Drug: Placebo; Drug: VYNT-0126

• Study Type: Interventional
• Enrollment (Anticipated): 48
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 43 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Female

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Liquid for oral administration once daily
Experimental: 5 mg VYNT-0126	Drug: VYNT-0126; Liquid for oral administration once daily
Experimental: 10 mg VYNT-0126	Drug: VYNT-0126; Liquid for oral administration once daily

What is the study measuring?

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	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 Outcome Measures

Outcome Measure	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	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).

Collaborators and Investigators

• Sponsor: Vyant Bio

Study record dates

Study Major Dates

• Study Start (Anticipated): March 1, 2023
• Primary Completion (Anticipated): March 1, 2024
• Study Completion (Anticipated): June 1, 2024

Study Registration Dates

• First Submitted: November 7, 2022
• First Submitted That Met QC Criteria: November 14, 2022
• First Posted (Actual): November 23, 2022

Study Record Updates

• Last Update Posted (Actual): November 23, 2022
• Last Update Submitted That Met QC Criteria: November 14, 2022
• Last Verified: November 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Syndrome; Rett Syndrome
• Other Study ID Numbers: VYNT-0126-001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No