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

Vyant Bio

Status and phase

Not yet enrolling

Phase 2

Conditions

Rett Syndrome

Treatments

Drug: Placebo

Drug: VYNT-0126

Study type

Interventional

Funder types

Industry

Identifiers

NCT05625568

VYNT-0126-001

Details and patient eligibility

About

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.

Enrollment

48 estimated patients

Sex

Female

Ages

18 to 45 years old

Volunteers

No 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