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

Rett Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05625568
• Other study ID #: VYNT-0126-001
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: March 2023
• Est. completion date: June 2024

Study information

• Verified date: November 2022
• Source: Vyant Bio
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 48
• Est. completion date: June 2024
• Est. primary completion date: March 2024
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 18 Years to 45 Years

Eligibility

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

Related Conditions & MeSH terms

• Rett Syndrome
• Syndrome

Intervention

Drug:
• VYNT-0126
Liquid for oral administration once daily
• Placebo
Liquid for oral administration once daily

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Vyant Bio

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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).
Other	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).
Primary	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	Change from Baseline in the Rett Syndrome Behavioral Questionnaire (RSBQ)		Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
Secondary	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).
Secondary	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).
Secondary	Clinical Global Impression of Improvement (CGI-I)		Obtained at the end of dose titration and end of treatment (approximately 14 weeks).