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The STARS Phase 2 Study: A Randomized Controlled Trial of Gaboxadol in Angelman Syndrome
Lynne M Bird1, Cesar Ochoa-Lubinoff1, Wen-Hann Tan1
1From the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston.
Gaboxadol (OV101) demonstrated a favorable safety profile and was well-tolerated in individuals with Angelman syndrome (AS). Exploratory analysis indicated potential efficacy, warranting further investigation in future studies.
Area of Science:
- Neuroscience
- Pharmacology
- Genetics
Background:
- Angelman syndrome (AS) is a rare neurodevelopmental disorder characterized by severe intellectual disability, motor deficits, and epilepsy.
- Current treatments for AS are limited, highlighting the need for novel therapeutic strategies.
- Gaboxadol, a selective GABAA receptor agonist, targets extrasynaptic receptors implicated in neuronal excitability.
Purpose of the Study:
- To assess the safety and tolerability of gaboxadol (OV101) in individuals with Angelman syndrome.
- To explore the preliminary efficacy of gaboxadol in AS patients.
- To compare gaboxadol treatment against a placebo in a controlled trial setting.
Main Methods:
- A multicenter, double-blind, placebo-controlled, parallel-group trial involving adolescent and adult patients with AS.
- Randomization to gaboxadol (10 mg or 15 mg twice daily) or placebo for 12 weeks.
- Safety monitoring and assessment of exploratory efficacy using adapted Clinical Global Impression scales.
Main Results:
- Eighty-seven participants received study drug, with 90% completing the 12-week trial.
- Adverse events were predominantly mild to moderate; no life-threatening events were reported.
- Gaboxadol showed statistically significant improvement on the AS-adapted CGI-I scale compared to placebo (p=0.0006).
Conclusions:
- Gaboxadol (OV101) was found to be safe and well-tolerated in individuals with Angelman syndrome over 12 weeks.
- The observed efficacy, though exploratory, suggests potential therapeutic benefits for AS.
- Further clinical studies are warranted to confirm the efficacy of gaboxadol in treating Angelman syndrome.
