Clinical trial NCT05625568: 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
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Rett Syndrome
Lead sponsor: Vyant Bio
Sponsor class: INDUSTRY
First posted: 2022-11-23
Enrollment: 48
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Adverse Events
timeFrame: Through study completion, approximately 14 weeks
Secondary outcomes:
measure: Change from Baseline in the Rett Syndrome Behavioral Questionnaire (RSBQ)
timeFrame: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
measure: Change from Baseline in the 24-Item Motor-Behavioral Assessment (MBA)
timeFrame: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
measure: Change from Baseline in Clinical Global Impression of Severity (CGI-S)
timeFrame: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
measure: Clinical Global Impression of Improvement (CGI-I)
timeFrame: Obtained at the end of dose titration and end of treatment (approximately 14 weeks).
Other outcomes:
measure: Quantitative Electroencephalograms (EEGs)
timeFrame: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
measure: Electrophysiological Evoked Potentials
timeFrame: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
Locations:
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