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

Phase 2

Not yet recruiting

• Conditions: Rett Syndrome
• Interventions: Drug: Placebo; Drug: VYNT-0126

• Registration Number: NCT05625568
• Lead Sponsor: Vyant Bio

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2022-11
• Study First Submitted: 2022-11-07
• Study First Submitted QC: 2022-11-14
• Last Update Submitted: 2022-11-14
• Study First Posted: 2022-11-23
• Last Update Posted: 2022-11-23
• Study Start: 2023-03
• Primary Completion: 2024-03
• Study Completion: 2024-06

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: Female
• Target Recruitment: 48

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	-
5 mg VYNT-0126	VYNT-0126	-
10 mg VYNT-0126	VYNT-0126	-

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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).