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

• ClinicalTrials.gov Identifier: NCT04106557
• Recruitment Status: Completed
• First Posted: September 27, 2019
• Last Update Posted: November 17, 2020
• Sponsor: Ovid Therapeutics Inc.
• Information provided by (Responsible Party): Ovid Therapeutics Inc.

Study Description

Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Primary Disease or Condition Being Studied: Angelman Syndrome (AS)	Drug: Gaboxadol; Drug: Placebo	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 104 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Masking Description: Double (Participant/Care Giver and Investigator/Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: 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
• Actual Study Start Date: September 9, 2019
• Actual Primary Completion Date: November 2, 2020
• Actual Study Completion Date: November 2, 2020

Arms and Interventions

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.

Outcome Measures

Primary Outcome Measures :

1. Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) [ Time Frame: 12 weeks ]
   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.

Eligibility Criteria

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

Criteria

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.

Contacts and Locations

Locations

United States, Arizona

Ovid Therapeutics Investigative Site

Phoenix, Arizona, United States, 85006

United States, California

Ovid Therapeutics Investigative Site

San Diego, California, United States, 92024

United States, Illinois

Ovid Therapeutics Investigative Site

Chicago, Illinois, United States, 60612

United States, Massachusetts

Ovid Therapeutics Investigative Site

Boston, Massachusetts, United States, 02115

Ovid Therapeutics Investigative Site

Lexington, Massachusetts, United States, 02421

United States, Ohio

Ovid Therapeutics Investigative Site

Cincinnati, Ohio, United States, 45229

United States, Pennsylvania

Ovid Therapeutics Investigative Site

Media, Pennsylvania, United States, 19063

United States, Tennessee

Ovid Therapeutics Investigative Site

Nashville, Tennessee, United States, 37232

United States, Washington

Ovid Therapeutics Investigative Site

Tacoma, Washington, United States, 85006

Australia, Queensland

Ovid Therapeutics Investigative Site

Brisbane, Queensland, Australia, 4101

Australia, Victoria

Ovid Therapeutics Investigative Site

Heidelberg, Victoria, Australia, 3084

Germany

Ovid Therapeutics Investigative Site

Munich, Germany, 80804

Israel

Ovid Therapeutics Investigative Site

Ramat Gan, Israel, 52621

Netherlands

Ovid Therapeutics Investigative Site

Rotterdam, Netherlands, 3012CN

Sponsors and Collaborators

Ovid Therapeutics Inc.

More Information

• Responsible Party: Ovid Therapeutics Inc.
• ClinicalTrials.gov Identifier: NCT04106557
• Other Study ID Numbers: OV101-19-001
• First Posted: September 27, 2019
• Last Update Posted: November 17, 2020
• Last Verified: November 2020

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Angelman Syndrome; Syndrome; Disease; Pathologic Processes; Movement Disorders; Central Nervous System Diseases; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Gaboxadol; Analgesics; Sensory System Agents; Peripheral Nervous System Agents; Physiological Effects of Drugs; Anticonvulsants; GABA Agonists; GABA Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action