A Study in Adults and Adolescents With Angelman Syndrome (STARS)

March 3, 2022 updated by: Ovid Therapeutics Inc.

A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol

The purpose of the study is to assess the safety and tolerability of oral OV101 (gaboxadol) in adult and adolescent subjects with Angelman syndrome. In addition, several exploratory efficacy outcome measures will be investigated.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Two dosing schedules of OV101 (gaboxadol) giving as once daily or twice daily dose will be assessed against placebo.

Study Type

Interventional

Enrollment (Actual)

88

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Israel
      • Ramat Gan, Israel
        • Ovid Therapeutics Investigative Site
  • United States
    • Arizona
      • Phoenix, Arizona, United States
        • Ovid Therapeutics Investigative Site
    • California
      • San Diego, California, United States
        • Ovid Therapeutics Investigative Site
    • Florida
      • Gainesville, Florida, United States
        • Ovid Therapeutics Investigative Site
      • Tampa, Florida, United States
        • Ovid Therapeutics Investigative Site
    • Georgia
      • Atlanta, Georgia, United States
        • Ovid Therapeutics Investigative Site
    • Illinois
      • Chicago, Illinois, United States
        • Ovid Therapeutics Investigative Site
    • Massachusetts
      • Boston, Massachusetts, United States
        • Ovid Therapeutics Investigative Site
      • Lexington, Massachusetts, United States
        • Ovid Therapeutics Investigative Site
    • Ohio
      • Cincinnati, Ohio, United States
        • Ovid Therapeutics Investigative Site
    • Pennsylvania
      • Media, Pennsylvania, United States
        • Ovid Therapeutics Investigative Site
    • South Carolina
      • Greenwood, South Carolina, United States
        • Ovid Therapeutics Investigative Site
    • Tennessee
      • Nashville, Tennessee, United States
        • Ovid Therapeutics Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

13 years to 49 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria

  1. Age 13- 49 years
  2. Diagnosis of Angelman syndrome
  3. Receiving a stable regimen of concomitant medications for at least 4 weeks prior to Baseline, and able to maintain these throughout the duration of the study
  4. Has a caregiver capable of providing informed consent on behalf of the subject and able to attend scheduled study visits
  5. Able to ingest study medication
  6. Caregivers must agree not to post any subject or study information on social media

Exclusion Criteria

  1. Unable to perform the study related safety and exploratory efficacy assessments, such as motor function
  2. Poorly controlled seizure activity
  3. Concomitant cardiovascular, respiratory, liver, renal, or hematologic diseases of a degree that would limit participation in the study
  4. Pregnancy or women of child-bearing potential who are not using and acceptable method of contraception
  5. Concomitant use of minocycline, levodopa, zolpidem, zaleplon, eszopiclone, ramelteon, and cannabinoid derivatives, or any other use of any investigational agent, device, and/or investigational procedure 4 weeks prior to Baseline and during the study
  6. Allergy to OV101 or any excipients
  7. At increased risk of harming self and/or others based on investigator assessment
  8. Any condition or reason that in the opinion of the investigator makes the subject unsuitable for enrollment
  9. Inability of subject or caregiver to comply with study requirements

Other protocol-defined inclusion/exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Twice daily
Other: Placebo
Experimental: OV101 regimen 1
OV101 once daily
Drug: OV101 Regimen 1
Other Names:
  • Gaboxadol
Experimental: OV101 regimen 2
OV101 twice daily
Drug: OV101 regimen 2
Other Names:
  • Gaboxadol

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of Adverse Events in Placebo and Active Treatment Groups
Time Frame: Baseline and Week 12
Summary of Subjects Reporting at least one Treatment Emergent Adverse Event (TEAEs), Safety Set. The table below summarizes the subjects who experienced TEAEs in the study.
Baseline and Week 12
Incidence of Adverse Events in Placebo and Active Treatment Groups
Time Frame: Baseline and Week 12
The Treatment Emergent Adverse Event (TEAEs) Reported by ≥10% of Subjects in Any Treatment Group by Preferred Term, Safety Set.
Baseline and Week 12
Incidence of Adverse Events in Placebo and Active Treatment Groups
Time Frame: Baseline and Week 12
Treatment-related TEAEs (Treatment Emergent Adverse Event) in ≥ 2 Subjects in OV101 Combined, Safety Set. The incidence of TEAEs assessed as treatment-related (at least possibly related to study drug, by the Investigator). Preferred Term in the table below.
Baseline and Week 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ovid Therapeutics Inc.

Investigators

  • Study Director: Amit Rakhit, MD, MBA, Ovid Therapeutics Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2016

Primary Completion (Actual)

June 7, 2018

Study Completion (Actual)

August 6, 2018

Study Registration Dates

First Submitted

December 2, 2016

First Submitted That Met QC Criteria

December 14, 2016

First Posted (Estimate)

December 19, 2016

Study Record Updates

Last Update Posted (Actual)

March 31, 2022

Last Update Submitted That Met QC Criteria

March 3, 2022

Last Verified

March 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • OV101-15-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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