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

NCT ID: NCT02996305

Last Updated: 2025-08-03

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

88 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-01-31

Study Completion Date

2018-08-06

Brief Summary

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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.

Detailed Description

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

Conditions

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Angelman Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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OV101 regimen 1

OV101 once daily

Group Type EXPERIMENTAL

OV101 Regimen 1

Intervention Type DRUG

OV101 regimen 2

OV101 twice daily

Group Type EXPERIMENTAL

OV101 regimen 2

Intervention Type DRUG

Placebo

Twice daily

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Interventions

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OV101 Regimen 1

Intervention Type DRUG

OV101 regimen 2

Intervention Type DRUG

Placebo

Intervention Type OTHER

Other Intervention Names

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Gaboxadol Gaboxadol

Eligibility Criteria

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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
Minimum Eligible Age

13 Years

Maximum Eligible Age

49 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Healx AI

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Amit Rakhit, MD, MBA

Role: STUDY_DIRECTOR

Healx AI

Locations

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Ovid Therapeutics Investigative Site

Phoenix, Arizona, United States

Site Status

Ovid Therapeutics Investigative Site

San Diego, California, United States

Site Status

Ovid Therapeutics Investigative Site

Gainesville, Florida, United States

Site Status

Ovid Therapeutics Investigative Site

Tampa, Florida, United States

Site Status

Ovid Therapeutics Investigative Site

Atlanta, Georgia, United States

Site Status

Ovid Therapeutics Investigative Site

Chicago, Illinois, United States

Site Status

Ovid Therapeutics Investigative Site

Boston, Massachusetts, United States

Site Status

Ovid Therapeutics Investigative Site

Lexington, Massachusetts, United States

Site Status

Ovid Therapeutics Investigative Site

Cincinnati, Ohio, United States

Site Status

Ovid Therapeutics Investigative Site

Media, Pennsylvania, United States

Site Status

Ovid Therapeutics Investigative Site

Greenwood, South Carolina, United States

Site Status

Ovid Therapeutics Investigative Site

Nashville, Tennessee, United States

Site Status

Ovid Therapeutics Investigative Site

Ramat Gan, , Israel

Site Status

Countries

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United States Israel

References

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Bird LM, Ochoa-Lubinoff C, Tan WH, Heimer G, Melmed RD, Rakhit A, Visootsak J, During MJ, Holcroft C, Burdine RD, Kolevzon A, Thibert RL. The STARS Phase 2 Study: A Randomized Controlled Trial of Gaboxadol in Angelman Syndrome. Neurology. 2021 Feb 16;96(7):e1024-e1035. doi: 10.1212/WNL.0000000000011409. Epub 2020 Dec 21.

Reference Type DERIVED
PMID: 33443117 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan: Study Protocol

View Document

Document Type: Statistical Analysis Plan: Statistical Analysis Plan document

View Document

Other Identifiers

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OV101-15-001

Identifier Type: -

Identifier Source: org_study_id

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