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Completed

NCT Number: NCT02996305

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

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.

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Key information

Conditions

Angelman Syndrome Abnormalities, Multiple Central Nervous System Diseases Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Imprinting Disorders Movement Disorders Nervous System Diseases

Age range

13 year–49 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Ovid Therapeutics Investigative Site, Ramat Gan, Israel

Loading trial locations.

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age 13- 49 years
  • Diagnosis of Angelman syndrome
  • 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
  • Has a caregiver capable of providing informed consent on behalf of the subject and able to attend scheduled study visits
  • Able to ingest study medication
  • Caregivers must agree not to post any subject or study information on social media

Exclusion criteria

  • Unable to perform the study related safety and exploratory efficacy assessments, such as motor function
  • Poorly controlled seizure activity
  • Concomitant cardiovascular, respiratory, liver, renal, or hematologic diseases of a degree that would limit participation in the study
  • Pregnancy or women of child-bearing potential who are not using and acceptable method of contraception
  • 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
  • Allergy to OV101 or any excipients
  • At increased risk of harming self and/or others based on investigator assessment
  • Any condition or reason that in the opinion of the investigator makes the subject unsuitable for enrollment
  • Inability of subject or caregiver to comply with study requirements

Other protocol-defined inclusion/exclusion criteria may apply.

Treatment and study plan

OV101 Regimen 1

Drug

Other names: Gaboxadol

OV101 regimen 2

Drug

Other names: Gaboxadol

Placebo

Other

Primary outcomes

  1. 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.

  2. 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.

  3. 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.

Sponsors and collaborators

Lead sponsor

Healx AI

Industry

Registry information

Official study title

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

Important dates

Study start
2016
Primary completion
2018
Study completion
2018
First posted
Dec 19, 2016
Registry last updated
Aug 3, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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