- Gaboxadol (OV101) shows benefit in several areas of Angelman syndrome (AS).
Why this matters
- AS is a genetic neurodevelopmental disorder that causes intellectual, speech, motor, behavior, and sleep problems, as well as seizures.
Gaboxadol was safe and tolerated. 78/88 (89%) patients completed the study.
Study discontinuations for adverse events (AEs): 1 receiving placebo, 3 receiving gaboxadol twice daily.
Most AEs were mild and similar between groups.
Efficacy was an exploratory endpoint.
- Daily gaboxadol showed improvements vs placebo (P=.0006). 67% rated “improved.”
- Improvements in sleep (P=.0141), gross motor function (P=.0522), and motor skills.
- Suggestions of improvements in communication, challenging behavior, and anxiety.
Phase 2, double-blinded, placebo-controlled trial.
Patients 13-49 years old randomly assigned to gaboxadol 15 mg daily, twice daily (10 mg then 15 mg), or placebo.
Commonly used medications for AS were allowed.
Patients evaluated using CGI-I and domain-specific instruments at 12 weeks.
Ovid Therapeutics sponsored the study.
- Actigraphy data on sleep latency and sleep time were difficult to obtain. Only a minority of patients tolerated the device.