Brief Summary

This open-label study (OV101-18-002) will evaluate the long-term (52 weeks) safety of OV101 in subjects with AS and provide additional OV101 treatment to those subjects who completed Study OV101-15-001 (NCT02996305). Subjects with AS who completed the pharmacokinetic Study OV101-16-001 (NCT03109756) will also be permitted to participate, provided they meet all entry criteria.

Trial Health

Monitor

Trial Health Score

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

Enrollment

141

participants targeted

Target at P25-P50 for phase_3

Timeline

Completed

Started Jan 2019

Geographic Reach

2 countries

11 active sites

Status

terminated

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

2.4 years study duration

Study Start

First participant enrolled

January 31, 2019

Completed

2 months until next milestone

First Submitted

Initial submission to the registry

March 18, 2019

Completed

2 days until next milestone

First Posted

Study publicly available on registry

March 20, 2019

Completed

2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 4, 2021

Completed

26 days until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2021

Completed

4.1 years until next milestone

Results Posted

Study results publicly available

July 18, 2025

Completed

Last Updated

July 18, 2025

Status Verified

July 1, 2025

Enrollment Period

2.3 years

First QC Date

March 18, 2019

Results QC Date

March 18, 2024

Last Update Submit

July 17, 2025

Conditions

Angelman Syndrome

Outcome Measures

Primary Outcomes (1)

Incidence of Participants Experiencing Adverse Events in Active Treatment Group
Safety assessments related to the primary study objective of evaluating the safety and tolerability of OV101, including serious adverse events (SAEs) and adverse events (AEs) leading to study discontinuation, as assessed by the number of participants who experienced at least one adverse event.
Change from baseline to Week 39

Secondary Outcomes (1)

To Evaluate the Long-term Efficacy of OV101 Treatment as Assessed by Changes in Behavior in Study Participants With AS Who Were at Least 4 Years Old.
change from baseline to 12 wks and baseline to early termination of the study at 39 weeks

Study Arms (1)

OV101

EXPERIMENTAL

once daily at bedtime (gaboxadol)

Drug: OV101

Interventions

OV101DRUG

Each subject will be titrated to his or her maximal tolerated daily dose, up to a maximum daily dose of 15 mg at bedtime.

Also known as: gaboxadol

OV101

Eligibility Criteria

Age13 Years - 49 Years

Sexall

Healthy VolunteersNo

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

You may qualify if:

Each subject must meet all the following criteria to be enrolled in this study:
Has completed the OV101-15-001 or OV101-16-001 study up to the EOS.
Has a previous diagnosis of AS with molecular confirmation from the OV101-15-001 or OV101-16-001 study.
Has an LAR/caregiver capable of providing informed consent and able to attend all scheduled study visits, oversee the administration of study drug, and provide feedback regarding the subject's symptoms and performance as described in the protocol.
Provides assent to the protocol (to the extent possible and in accordance with local institutional review board (IRB) and regulatory requirements) and has an LAR/caregiver who will provide written informed consent. Subjects providing assent must do so at the same visit as LAR/caregiver written informed consent is provided.
Can swallow study drug capsules or ingest the contents of study drug capsules after sprinkling the capsule contents onto 1 spoon of applesauce or low-fat yogurt.
Is currently receiving a stable dose of concomitant medications such as anti-epileptic medication, gabapentin, clonidine, trazadone, melatonin, and special diets for at least 4 weeks prior to Baseline.
Agrees to remain sexually abstinent from the first day of screening until 30 days after the last dose of study treatment.
Has LAR/caregiver(s) who agree not to post any of the subject's personal medical data or information related to the study on any website or social media site (eg, Facebook, Instagram, Twitter) until notified that the study is completed.

You may not qualify if:

Subjects meeting any of the following criteria will be excluded from the study:
Discontinued from the OV101-15-001 or OV101-16-001 study due to safety reasons causally related to OV101.
Has a concomitant disease (eg, gastrointestinal, renal, hepatic, endocrine, respiratory, or cardiovascular system disease) or condition or any clinically significant finding at Screening that could interfere with the conduct of the study or that would pose an unacceptable risk to the subject in this study.
Has poorly controlled seizures defined as \> 3 seizures lasting \< 3 minutes per week or \> 1 seizure episode lasting more than 3 minutes per week or as per medical monitor judgment.
Has clinically significant clinical laboratory abnormalities or vital signs at the time of screening (eg, alanine aminotransferase or aspartate aminotransferase \> 2.5 × upper limit of normal; total bilirubin or creatinine \> 1.5 × upper limit of normal). Retesting of clinical laboratory parameters may be allowed after consultation with the medical monitor or designee.
Current use of benzodiazepines, zolpidem, zaleplon, zopiclone, eszopiclone, barbiturates, or ramelteon for sleep within the 4 weeks prior to Day 1. Benzodiazepines administered for situational anxiety related to occasional procedures or events are permitted.
Has a history of suicidal behavior or is considered by the investigator to be at increased risk of suicide.
Has any condition or circumstance that, in the opinion of the investigator, makes the subject unsuitable for enrollment.
Has enrolled in any clinical trial or used any investigational agent or device, or has participated in any investigational procedure, within the 30 days before screening or does so concurrently with this study.
Is a family member of the investigator or of study site staff.

Contact the study team to confirm eligibility.