NCT02996305

A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol

Completed Phase 2 Results posted Last updated 17 July 2025

What this trial tests

Phase 2 trial testing OV101 Regimen 1 in Angelman Syndrome in 88 participants. Completed in 6 August 2018.

Timeline

1 January 2016

Primary endpoint
7 June 2018

6 August 2018

Quick facts

Lead sponsor	Healx AI
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	88
Start date	1 January 2016
Primary completion	7 June 2018
Estimated completion	6 August 2018
Sites	13 locations across United States, Israel

Drugs / interventions tested

• OV101 Regimen 1 — full drug profile →
• OV101 regimen 2 — full drug profile →
• Placebo

Conditions studied

• Angelman Syndrome — all drugs for Angelman Syndrome →

Sponsor

Healx AI — full company profile →

Who can join

Adults 13 to 49, any sex, with Angelman Syndrome. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Incidence of Adverse Events in Placebo and Active Treatment Groups
  Time frame: Baseline and Week 12
  Summary of Subjects Reporting at least one Treatment Emergent Adverse Event (TEAEs), Safety Set. The table below summarizes the subjects who experienced TEAEs in the study.
• Incidence of Adverse Events in Placebo and Active Treatment Groups
  Time frame: Baseline and Week 12
  The Treatment Emergent Adverse Event (TEAEs) Reported by ≥10% of Subjects in Any Treatment Group by Preferred Term, Safety Set.
• Incidence of Adverse Events in Placebo and Active Treatment Groups
  Time frame: Baseline and Week 12
  Treatment-related TEAEs (Treatment Emergent Adverse Event) in ≥ 2 Subjects in OV101 Combined, Safety Set. The incidence of TEAEs assessed as treatment-related (at least possibly related to study drug, by the Investigator). Preferred Term in the table below.

Sponsor's own description

The purpose of the study is to assess the safety and tolerability of oral OV101 (gaboxadol) in adult and adolescent subjects with Angelman syndrome. In addition, several exploratory efficacy outcome measures will be investigated.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Decreased surface expression of the δ subunit of the GABA_A receptor contributes to reduced tonic inhibition in dentate granule cells in a mouse model of fragile X syndrome.
   Zhang N, Peng Z, Tong X, Lindemeyer AK, et al · · 2017 · cited 39× · PMID 28822839 · DOI 10.1016/j.expneurol.2017.08.008
2. Hypothesis-driven investigations of diverse pharmacological targets in two mouse models of autism.
   Rhine MA, Parrott JM, Schultz MN, Kazdoba TM, et al · · 2019 · cited 32× · PMID 30653853 · DOI 10.1002/aur.2066
3. The STARS Phase 2 Study: A Randomized Controlled Trial of Gaboxadol in Angelman Syndrome.
   Bird LM, Ochoa-Lubinoff C, Tan WH, Heimer G, et al · · 2021 · cited 23× · PMID 33443117 · DOI 10.1212/wnl.0000000000011409
4. Development of an adapted Clinical Global Impression scale for use in Angelman syndrome.
   Kolevzon A, Ventola P, Keary CJ, Heimer G, et al · · 2021 · cited 17× · PMID 33397286 · DOI 10.1186/s11689-020-09349-8
5. Discovery of a new class of orthosteric antagonists with nanomolar potency at extrasynaptic GABA_A receptors.
   Falk-Petersen CB, Tsonkov TM, Nielsen MS, Harpsøe K, et al · · 2020 · cited 9× · PMID 32572053 · DOI 10.1038/s41598-020-66821-0
6. Promising therapeutic aspects in human genetic imprinting disorders.
   Chao Y, Qin Y, Zou X, Wang X, et al · · 2022 · cited 6× · PMID 36371218 · DOI 10.1186/s13148-022-01369-6