AAN 2019—Angelman syndrome: Phase 2 trial shows benefit of gaboxadol


  • Daniel M. Keller, Ph.D
  • Conference Reports
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Takeaway

  • 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.

Key results

  • 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.

Study design  

  • 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.

Limitations

  • Actigraphy data on sleep latency and sleep time were difficult to obtain. Only a minority of patients tolerated the device.