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

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.

Study Overview

• Status: Completed
• Conditions: Primary Disease or Condition Being Studied: Angelman Syndrome (AS)
• Intervention / Treatment: Drug: Gaboxadol; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 104
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Queensland
    • Brisbane, Queensland, Australia, 4101
      • Ovid Therapeutics Investigative Site
  • Victoria
    • Heidelberg, Victoria, Australia, 3084
      • Ovid Therapeutics Investigative Site
• Germany
  • Munich, Germany, 80804
    • Ovid Therapeutics Investigative Site
• Israel
  • Ramat Gan, Israel, 52621
    • Ovid Therapeutics Investigative Site
• Netherlands
  • Rotterdam, Netherlands, 3012CN
    • Ovid Therapeutics Investigative Site
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85006
      • Ovid Therapeutics Investigative Site
  • California
    • San Diego, California, United States, 92024
      • Ovid Therapeutics Investigative Site
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Ovid Therapeutics Investigative Site
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Ovid Therapeutics Investigative Site
    • Lexington, Massachusetts, United States, 02421
      • Ovid Therapeutics Investigative Site
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Ovid Therapeutics Investigative Site
  • Pennsylvania
    • Media, Pennsylvania, United States, 19063
      • Ovid Therapeutics Investigative Site
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Ovid Therapeutics Investigative Site
  • Washington
    • Tacoma, Washington, United States, 85006
      • Ovid Therapeutics Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 12 years (Child)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 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).	12 weeks

Collaborators and Investigators

• Sponsor: Ovid Therapeutics Inc.

Study record dates

Study Major Dates

• Study Start (Actual): September 9, 2019
• Primary Completion (Actual): November 2, 2020
• Study Completion (Actual): November 2, 2020

Study Registration Dates

• First Submitted: September 25, 2019
• First Submitted That Met QC Criteria: September 25, 2019
• First Posted (Actual): September 27, 2019

Study Record Updates

• Last Update Posted (Estimated): January 8, 2024
• Last Update Submitted That Met QC Criteria: January 5, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Central Nervous System Diseases; Nervous System Diseases; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Movement Disorders; Abnormalities, Multiple; Chromosome Disorders; Syndrome; Angelman Syndrome; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; Analgesics; Sensory System Agents; GABA Agents; Anticonvulsants; GABA Agonists; Gaboxadol
• Other Study ID Numbers: OV101-19-001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No