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

Angelman Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02996305
• Other study ID #: OV101-15-001
• Status: Completed
• Phase: Phase 2
• Start date: January 2016
• Est. completion date: August 6, 2018

Study information

• Verified date: March 2022
• Source: Ovid Therapeutics Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 88
• Est. completion date: August 6, 2018
• Est. primary completion date: June 7, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 13 Years to 49 Years

Eligibility

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.

Related Conditions & MeSH terms

• Angelman Syndrome
• Syndrome

Intervention

Drug:
• OV101 Regimen 1

• OV101 regimen 2

Other:
• Placebo

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Ovid Therapeutics Inc.

Countries where clinical trial is conducted

United States,  Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Baseline and Week 12
Primary	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.	Baseline and Week 12
Primary	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.	Baseline and Week 12