Johns Hopkins registers Epidiolex pilot trial for presymptomatic Sturge-Weber, Jazz as collaborator

The week's sole item concerns a Johns Hopkins University-sponsored trial of Jazz Pharmaceuticals' Epidiolex in infants with Sturge-Weber syndrome.

• The trial, "Epi-Pre" (NCT07778810), is an open-label, phase 2 dose escalation study sponsored by Johns Hopkins University, with Jazz Pharmaceuticals and the Hugo W. Moser Research Institute at Kennedy Krieger, Inc. listed as collaborators.¹ The study is currently recruiting, with an estimated primary completion date of July 2028. It will enroll infants aged 1 to 18 months with Sturge-Weber syndrome who have no history of seizures¹, testing whether Epidiolex in presymptomatic Sturge-Weber patients may delay the onset of seizures and improve neurological outcome.¹ The plan calls for consenting up to 10 babies with SWS, with an initial funded phase of 6 subjects and an interim analysis before enrolling up to 4 more.¹ Dosing is described as starting at 2.5 mg/kg/day divided twice daily, with the source giving differing escalation timelines: the detailed description states weekly increases over the next 4 weeks to a maximum dose of 20 mg/kg/day¹, while the arms and interventions sections state increases over the next 5 weeks to a target dose of 20 mg/kg/day.¹