A Study of OV101 in Individuals With Angelman Syndrome (AS)

Phase 3 Completed 104 enrolled Healx AI

RandomizedParallel-groupDouble-blindTreatment

Summary

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

Timeline

Start: 2019-09-09
Primary completion: 2020-11-02
Completion: 2020-11-02

Outcome

Missed primary endpoint

paper There was no significant difference in CGI-I-AS between gaboxadol and placebo after 12 weeks of study treatment in pediatric AS participants. PMID 37639777 ↗

Publications

Keary C, Bird LM, de Wit MC, Hatti S, Heimer G, Heussler H, Kolevzon A, Mathews A, Ochoa-Lubinoff C, Tan WH, Yan Y, Adams M. Gaboxadol in angelman syndrome: A double-blind, parallel-group, randomized placebo-controlled phase 3 study. Eur J Paediatr Neurol. 2023 Nov;47:6-12. doi: 10.1016/j.ejpn.2023.07.008. Epub 2023 Aug 1.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Gaboxadol	Unknown	0.125 mg/kg	Oral
Subject	Gaboxadol	Unknown	0.24 mg/kg	Oral