Clinical trial NCT02996305: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Angelman Syndrome
Lead sponsor: Ovid Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2016-12-19
Enrollment: 88
Sex: ALL
Minimum age (years): 13
Maximum age (years): 49
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Incidence of Adverse Events in Placebo and Active Treatment Groups
timeFrame: Baseline and Week 12
measure: Incidence of Adverse Events in Placebo and Active Treatment Groups
timeFrame: Baseline and Week 12
measure: Incidence of Adverse Events in Placebo and Active Treatment Groups
timeFrame: Baseline and Week 12
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Ovid Therapeutics Investigative Site
state: Arizona
city: San Diego
country: United States
facility: Ovid Therapeutics Investigative Site
state: California
city: Gainesville
country: United States
facility: Ovid Therapeutics Investigative Site
state: Florida
city: Tampa
country: United States
facility: Ovid Therapeutics Investigative Site
state: Florida
city: Atlanta
country: United States
facility: Ovid Therapeutics Investigative Site
state: Georgia
city: Chicago
country: United States
facility: Ovid Therapeutics Investigative Site
state: Illinois
city: Boston
country: United States
facility: Ovid Therapeutics Investigative Site
state: Massachusetts
city: Lexington
country: United States
facility: Ovid Therapeutics Investigative Site
state: Massachusetts
city: Cincinnati
country: United States
facility: Ovid Therapeutics Investigative Site
state: Ohio
city: Media
country: United States
facility: Ovid Therapeutics Investigative Site
state: Pennsylvania
city: Greenwood
country: United States
facility: Ovid Therapeutics Investigative Site
state: South Carolina
city: Nashville
country: United States
facility: Ovid Therapeutics Investigative Site
state: Tennessee
city: Ramat Gan
country: Israel
facility: Ovid Therapeutics Investigative Site
Study officials:
affiliation: Ovid Therapeutics Inc.
name: Amit Rakhit, MD, MBA
role: STUDY_DIRECTOR
References:
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.