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

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

Official Title
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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

Conditions
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Primary Disease or Condition Being Studied: Angelman Syndrome (AS)

Intervention / Treatment
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* Drug: Gaboxadol
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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: Subjects 2 to 3 years old must have a minimum body weight of 9 kg. 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.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: OV101 once daily (weight-based dosing) Other Name:Gaboxadol<br>OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration | Drug: Gaboxadol<br>* OV101 versus placebo once daily at bedtime for 12 weeks<br>|
| Placebo Comparator: Placebo once daily<br>Matching placebo,oral, provided once daily at bedtime for 12 week duration | Drug: Placebo<br>* Matching placebo capsules to OV101 capsules.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) | Clinical Global Impressions-Improvement-Angelman Syndrome (CGI-I-AS) - Scale of 1-7 reflects a spectrum where 1 is most improved, 4 is no change, and 7 is most worsened. | 12 weeks |