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

NCT ID: NCT05625568

Last Updated: 2022-11-23

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 48 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-03-31
• Study Completion Date: 2024-06-30

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.

Conditions

Rett Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Liquid for oral administration once daily

5 mg VYNT-0126

Group Type: EXPERIMENTAL

VYNT-0126

Intervention Type: DRUG

Liquid for oral administration once daily

10 mg VYNT-0126

Group Type: EXPERIMENTAL

VYNT-0126

Intervention Type: DRUG

Liquid for oral administration once daily

Interventions

VYNT-0126

Liquid for oral administration once daily

Intervention Type: DRUG

Placebo

Liquid for oral administration once daily

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 45 Years
• Eligible Sex: FEMALE
• Accepts Healthy Volunteers: No

Sponsors

Vyant Bio

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Other Identifiers

VYNT-0126-001

Identifier Type: -

Identifier Source: org_study_id