Ovid Therapeutics is recruiting adult patients with Angelman syndrome to a Phase 2 clinical trial of gaboxadol, a compound that aims to treat the disease by restoring lost inhibitory brain signaling.
The study, which is running in a number of U.S. clinics and Israel, aims to recruit 75 patients to assess the safety and effectiveness of the compound, which also is in development for Fragile X syndrome.
To participate in the study, called STARS (NCT02996305), patients need to have a caregiver who can provide consent, attend scheduled visits, including phone meetings, and participate in study assessments.
The Phase 2 STARS study will explore once or twice daily dosing of gaboxadol. Patients will be assigned randomly to one of the gaboxadol treatment groups or a placebo in a double-blind manner. This means that neither patients nor study staff are aware of each patient’s treatment.
Gaboxadol, also called OV101 is a compound stimulating GABAA receptors that contain a delta subunit. Such receptors are crucial for a feature called tonic inhibition — a constant brake on other neurons that allow filtering of sensory information and neuronal activity.
The gene defect in Angelman, affecting the UBE3A gene, causes molecules that remove GABA after it has bound to its receptor to be overly active. This causes a removal of too much GABA, essentially removing the brake on other neurons. Researchers believe that this is one of the core mechanisms leading to the developmental abnormalities in Angelman syndrome.
Gaboxadol does not impact the removal of GABA. Instead, it activates the GABAA receptor on its own to restore the neuronal brake.
Ovid says that gaboxadol improves walking capacity and balance in mice models of Angelman, and in October, the company presented data showing that it improved behavioral abnormalities in a Fragile X mouse model.
In humans, an ongoing Phase 1 clinical trial (NCT03109756) is exploring how a single dose of gaboxadol impacts young patients — aged 13 to 17 — with Angelman or Fragile X syndrome. The aim of the study is to pick a gaboxadol dose to use in future trials.
Caregivers and patients interested in participating in the trial can read more about it at the trial webpage or at its registration page in the clinical trials database, where contact details and study locations can also be found.