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

• Status: Not yet recruiting
• Trial end date: 2024-06-01
• Target enrollment: 0
• Participant gender: Female

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vyant Bio

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