Phase 12 Study of ION283 Injected into the Spine for Treating Lafora Disease in Children and Teens

What this Trial Is About

Researchers are evaluating the safety and effectiveness of ION283, an anti-sense oligonucleotide therapy given by injection into the spinal fluid through a lumbar puncture, in patients aged 10 to 18 years with Lafora disease. This open-label Phase 12 study aims to understand how well multiple doses of 15 mg ION283 work and how safe they are for these patients over a period of two years. Participants will receive an initial dose of 15 mg ION283 as an intrathecal bolus injection every 12 weeks during the 24-month open-label treatment period, following a 4-week screening period. All enrolled subjects will receive the same dose and treatment without placebo or comparison groups. Throughout the study, participants will undergo assessments including clinical evaluations, seizure frequency monitoring, and several quality of life and disability scales at baseline and after two years. Safety will be closely monitored by tracking treatment-related adverse events, and various EEG recordings will be used to assess changes in brain activity. Participants are expected to comply with study requirements for up to two years.

A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease