A Study of OV101 in Individuals With Angelman Syndrome (AS)

A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in Pediatric Individuals With Angelman Syndrome

The purpose of this study is to assess the efficacy and safety of oral OV101 (gaboxadol) in pediatric subjects with Angelman syndrome.

Inclusion Criteria:

• Male or female and 2 to 12 years old (inclusive) at the time of informed consent

• Confirmed molecular diagnosis of AS

• Has a CGI-S-AS score of 3 or more at baseline.

• Meets the following age-appropriate body weight criterion:

  1. Subjects 2 to 3 years old must have a minimum body weight of 9 kg.
  2. Subjects 4 years and older must be between 17 kg and 64 kg (inclusive).

• Stable concomitant mediations for at least 4 weeks before study start

Exclusion Criteria:

• Any condition that would limit study participation
• Clinically significant lab or vital sign abnormalities at the time of screening
• Poorly controlled seizures (weekly seizures of any frequency with a duration more than 3 minutes, weekly seizures occurring more than 3 times per week, each with a duration of less than 3 minutes, or as defined by investigator assessment)
• Use of prescription medications for sleep, minocycline, or levodopa within the 4 weeks prior to Day 1 or during the study. Benzodiazepines chronically administered for seizure control are permitted.
• Cannot comply with protocol study assessments during screening or caregiver unable to comply with study requirements.
• Enrolled in any clinical trial or used any investigational agent within the 30 days before screening or concurrently with this study.