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

Phase 3

Completed

• Conditions: Primary Disease or Condition Being Studied: Angelman Syndrome (AS)
• Interventions: Drug: Placebo; Drug: Gaboxadol

• Registration Number: NCT04106557
• Lead Sponsor: Ovid Therapeutics Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2019-09-09
• Study First Submitted: 2019-09-25
• Study First Submitted QC: 2019-09-25
• Study First Posted: 2019-09-27
• Primary Completion: 2020-11-02
• Study Completion: 2020-11-02
• Results First Submitted: 2022-11-15
• Status Verified: 2024-01
• Results First Submitted QC: 2024-01-05
• Last Update Submitted: 2024-01-05
• Results First Posted: 2024-01-08
• Last Update Posted: 2024-01-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 104

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo once daily	Placebo	Matching placebo,oral, provided once daily at bedtime for 12 week duration
OV101 once daily (weight-based dosing) Other Name:Gaboxadol	Gaboxadol	OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration

Primary Outcome Measures

Name	Time	Method
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	12 weeks	To evaluate the efficacy of OV101 versus placebo as assessed by the Clinical Global Impressions-Improvement-Angelman syndrome (CGI-I-AS) score at Week 12. CGIA-I-AS scores range from 1 (very much improved), to 4 (no change), to 7 (very much worse).

Trial Locations

Locations (1)

Ovid Therapeutics Investigative Site; 🇳🇱 Rotterdam, Netherlands