A Study in Adults and Adolescents With Angelman Syndrome (STARS)
Primary Purpose
Angelman Syndrome
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
OV101 Regimen 1
OV101 regimen 2
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Angelman Syndrome
Eligibility Criteria
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.
Sites / Locations
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Placebo Comparator
Arm Label
OV101 regimen 1
OV101 regimen 2
Placebo
Arm Description
OV101 once daily
OV101 twice daily
Twice daily
Outcomes
Primary Outcome Measures
Incidence of Adverse Events in Placebo and Active Treatment Groups
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.
Incidence of Adverse Events in Placebo and Active Treatment Groups
The Treatment Emergent Adverse Event (TEAEs) Reported by ≥10% of Subjects in Any Treatment Group by Preferred Term, Safety Set.
Incidence of Adverse Events in Placebo and Active Treatment Groups
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.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02996305
Brief Title
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
Official Title
A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol
Study Type
Interventional
2. Study Status
Record Verification Date
March 2022
Overall Recruitment Status
Completed
Study Start Date
January 2016 (undefined)
Primary Completion Date
June 7, 2018 (Actual)
Study Completion Date
August 6, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ovid Therapeutics Inc.
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
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
Two dosing schedules of OV101 (gaboxadol) giving as once daily or twice daily dose will be assessed against placebo.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Angelman Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
88 (Actual)
8. Arms, Groups, and Interventions
Arm Title
OV101 regimen 1
Arm Type
Experimental
Arm Description
OV101 once daily
Arm Title
OV101 regimen 2
Arm Type
Experimental
Arm Description
OV101 twice daily
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Twice daily
Intervention Type
Drug
Intervention Name(s)
OV101 Regimen 1
Other Intervention Name(s)
Gaboxadol
Intervention Type
Drug
Intervention Name(s)
OV101 regimen 2
Other Intervention Name(s)
Gaboxadol
Intervention Type
Other
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Incidence of Adverse Events in Placebo and Active Treatment Groups
Description
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.
Time Frame
Baseline and Week 12
Title
Incidence of Adverse Events in Placebo and Active Treatment Groups
Description
The Treatment Emergent Adverse Event (TEAEs) Reported by ≥10% of Subjects in Any Treatment Group by Preferred Term, Safety Set.
Time Frame
Baseline and Week 12
Title
Incidence of Adverse Events in Placebo and Active Treatment Groups
Description
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.
Time Frame
Baseline and Week 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
13 Years
Maximum Age & Unit of Time
49 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Amit Rakhit, MD, MBA
Organizational Affiliation
Ovid Therapeutics Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Ovid Therapeutics Investigative Site
City
Phoenix
State/Province
Arizona
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
San Diego
State/Province
California
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Gainesville
State/Province
Florida
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Tampa
State/Province
Florida
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Atlanta
State/Province
Georgia
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Chicago
State/Province
Illinois
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Boston
State/Province
Massachusetts
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Lexington
State/Province
Massachusetts
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Cincinnati
State/Province
Ohio
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Media
State/Province
Pennsylvania
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Greenwood
State/Province
South Carolina
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Nashville
State/Province
Tennessee
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Ramat Gan
Country
Israel
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
33443117
Citation
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.
Results Reference
derived
Learn more about this trial
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
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