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

Primary Purpose

Rett Syndrome

Status
Not yet recruiting
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
VYNT-0126
Placebo
Sponsored by
Vyant Bio
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Rett Syndrome

Eligibility Criteria

18 Years - 45 Years (Adult)FemaleDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm Type

    Placebo Comparator

    Experimental

    Experimental

    Arm Label

    Placebo

    5 mg VYNT-0126

    10 mg VYNT-0126

    Arm Description

    Outcomes

    Primary Outcome Measures

    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.

    Secondary Outcome Measures

    Change from Baseline in the Rett Syndrome Behavioral Questionnaire (RSBQ)
    Change from Baseline in the 24-Item Motor-Behavioral Assessment (MBA)
    Change from Baseline in Clinical Global Impression of Severity (CGI-S)
    Clinical Global Impression of Improvement (CGI-I)

    Full Information

    First Posted
    November 7, 2022
    Last Updated
    November 14, 2022
    Sponsor
    Vyant Bio
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05625568
    Brief Title
    Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Patients
    Official Title
    Phase 2, Double-Blind, Randomized, Placebo-Controlled Clinical Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Female Patients
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    November 2022
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    March 2023 (Anticipated)
    Primary Completion Date
    March 2024 (Anticipated)
    Study Completion Date
    June 2024 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Vyant Bio

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    Yes
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    No

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Rett Syndrome

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Parallel Assignment
    Masking
    ParticipantCare ProviderInvestigatorOutcomes Assessor
    Allocation
    Randomized
    Enrollment
    48 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Placebo
    Arm Type
    Placebo Comparator
    Arm Title
    5 mg VYNT-0126
    Arm Type
    Experimental
    Arm Title
    10 mg VYNT-0126
    Arm Type
    Experimental
    Intervention Type
    Drug
    Intervention Name(s)
    VYNT-0126
    Intervention Description
    Liquid for oral administration once daily
    Intervention Type
    Drug
    Intervention Name(s)
    Placebo
    Intervention Description
    Liquid for oral administration once daily
    Primary Outcome Measure Information:
    Title
    Adverse Events
    Description
    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.
    Time Frame
    Through study completion, approximately 14 weeks
    Secondary Outcome Measure Information:
    Title
    Change from Baseline in the Rett Syndrome Behavioral Questionnaire (RSBQ)
    Time Frame
    Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
    Title
    Change from Baseline in the 24-Item Motor-Behavioral Assessment (MBA)
    Time Frame
    Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
    Title
    Change from Baseline in Clinical Global Impression of Severity (CGI-S)
    Time Frame
    Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
    Title
    Clinical Global Impression of Improvement (CGI-I)
    Time Frame
    Obtained at the end of dose titration and end of treatment (approximately 14 weeks).
    Other Pre-specified Outcome Measures:
    Title
    Quantitative Electroencephalograms (EEGs)
    Description
    Quantitative EEGs will be explored as potential biomarkers of intervention effects on brain function and clinical severity.
    Time Frame
    Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
    Title
    Electrophysiological Evoked Potentials
    Description
    Evoked potential following auditory and visual stimuli will be explored as potential biomarkers of intervention effects on brain function and clinical severity.
    Time Frame
    Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).

    10. Eligibility

    Sex
    Female
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    45 Years
    Accepts Healthy Volunteers
    No
    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

    12. IPD Sharing Statement

    Learn more about this trial

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

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