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

NCT05625568 | Unknown Phase 2 FDA Drug

• 1 other identifier: VYNT-0126-001
• Study Type: interventional
• Target: 48
• Locations: 0 countries
• Sites: N/A

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• 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 Outcomes (2)

• Quantitative Electroencephalograms (EEGs)

  Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).

• Electrophysiological Evoked Potentials

  Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).

Study Arms (3)

Placebo

PLACEBO COMPARATOR

Drug: Placebo

5 mg VYNT-0126

EXPERIMENTAL

Drug: VYNT-0126

10 mg VYNT-0126

EXPERIMENTAL

Drug: VYNT-0126

Interventions

VYNT-0126 DRUG

Liquid for oral administration once daily

10 mg VYNT-0126; 5 mg VYNT-0126

Placebo DRUG

Liquid for oral administration once daily

Placebo

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Vyant Bio: lead

MeSH Terms

Conditions

Rett Syndrome

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 7, 2022
• First Posted: November 23, 2022
• Study Start: March 1, 2023
• Primary Completion: March 1, 2024
• Study Completion: June 1, 2024
• Last Updated: November 23, 2022

Record last verified: 2022-11