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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at P50-P75 for phase_2

Timeline

Completed

Started Jan 2016

Geographic Reach

2 countries

13 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

2.6 years study duration

Study Start

First participant enrolled

January 1, 2016

Completed

11 months until next milestone

First Submitted

Initial submission to the registry

December 2, 2016

Completed

17 days until next milestone

First Posted

Study publicly available on registry

December 19, 2016

Completed

1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 7, 2018

Completed

2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 6, 2018

Completed

3.7 years until next milestone

Results Posted

Study results publicly available

March 31, 2022

Completed

Last Updated

August 3, 2025

Status Verified

July 1, 2025

Enrollment Period

2.4 years

First QC Date

December 2, 2016

Results QC Date

March 3, 2022

Last Update Submit

July 17, 2025

Conditions

Angelman Syndrome

Outcome Measures

Primary Outcomes (3)

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

Study Arms (3)

OV101 regimen 1

EXPERIMENTAL

OV101 once daily

Drug: OV101 Regimen 1

OV101 regimen 2

EXPERIMENTAL

OV101 twice daily

Drug: OV101 regimen 2

Placebo

PLACEBO COMPARATOR

Twice daily

Other: Placebo

Interventions

OV101 Regimen 1DRUG

Also known as: Gaboxadol

OV101 regimen 1

OV101 regimen 2DRUG

Also known as: Gaboxadol

OV101 regimen 2

PlaceboOTHER

Placebo

Eligibility Criteria

Age13 Years - 49 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

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

Contact the study team to confirm eligibility.