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

Study Overview
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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.

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

Conditions
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Angelman Syndrome

Intervention / Treatment
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* Drug: OV101 Regimen 1
* Drug: OV101 regimen 2
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 13 Years
Maximum Age: 49 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: OV101 regimen 1<br>OV101 once daily | Drug: OV101 Regimen 1<br> <br> * Other names: Gaboxadol;|
| Experimental: OV101 regimen 2<br>OV101 twice daily | Drug: OV101 regimen 2<br> <br> * Other names: Gaboxadol;|
| Placebo Comparator: Placebo<br>Twice daily | Other: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| 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 | 
| 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 |