Brief Summary

The purpose of this study is to assess the efficacy and safety of oral OV101 (gaboxadol) in pediatric subjects with Angelman syndrome.

Trial Health

On Track

Trial Health Score

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

Enrollment

104

participants targeted

Target at P25-P50 for phase_3

Timeline

Completed

Started Sep 2019

Shorter than P25 for phase_3

Geographic Reach

5 countries

14 active sites

Status

completed

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

Trial Relationships

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Study Timeline

Key milestones and dates

1.2 years study duration

Study Start

First participant enrolled

September 9, 2019

Completed

16 days until next milestone

First Submitted

Initial submission to the registry

September 25, 2019

Completed

2 days until next milestone

First Posted

Study publicly available on registry

September 27, 2019

Completed

1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 2, 2020

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 2, 2020

Completed

3.2 years until next milestone

Results Posted

Study results publicly available

January 8, 2024

Completed

Last Updated

January 8, 2024

Status Verified

January 1, 2024

Enrollment Period

1.2 years

First QC Date

September 25, 2019

Results QC Date

November 15, 2022

Last Update Submit

January 5, 2024

Conditions

Primary Disease or Condition Being Studied: Angelman Syndrome (AS)

Outcome Measures

Primary Outcomes (1)

Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
To evaluate the efficacy of OV101 versus placebo as assessed by the Clinical Global Impressions-Improvement-Angelman syndrome (CGI-I-AS) score at Week 12. CGIA-I-AS scores range from 1 (very much improved), to 4 (no change), to 7 (very much worse).
12 weeks

Study Arms (2)

OV101 once daily (weight-based dosing) Other Name:Gaboxadol

EXPERIMENTAL

OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration

Drug: Gaboxadol

Placebo once daily

PLACEBO COMPARATOR

Matching placebo,oral, provided once daily at bedtime for 12 week duration

Drug: Placebo

Interventions

GaboxadolDRUG

OV101 versus placebo once daily at bedtime for 12 weeks

OV101 once daily (weight-based dosing) Other Name:Gaboxadol

PlaceboDRUG

Matching placebo capsules to OV101 capsules.

Placebo once daily

Eligibility Criteria

Age2 Years - 12 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

Male or female and 2 to 12 years old (inclusive) at the time of informed consent
Confirmed molecular diagnosis of AS
Has a CGI-S-AS score of 3 or more at baseline.
Meets the following age-appropriate body weight criterion:
Subjects 2 to 3 years old must have a minimum body weight of 9 kg.
Subjects 4 years and older must be between 17 kg and 64 kg (inclusive).
Stable concomitant mediations for at least 4 weeks before study start

You may not qualify if:

Any condition that would limit study participation
Clinically significant lab or vital sign abnormalities at the time of screening
Poorly controlled seizures (weekly seizures of any frequency with a duration more than 3 minutes, weekly seizures occurring more than 3 times per week, each with a duration of less than 3 minutes, or as defined by investigator assessment)
Use of prescription medications for sleep, minocycline, or levodopa within the 4 weeks prior to Day 1 or during the study. Benzodiazepines chronically administered for seizure control are permitted.
Cannot comply with protocol study assessments during screening or caregiver unable to comply with study requirements.
Enrolled in any clinical trial or used any investigational agent within the 30 days before screening or concurrently with this study.

Contact the study team to confirm eligibility.