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

Summary

This study will test the safety and efficacy of multiple doses of ION283 administered as intrathecal (IT) injections by lumbar puncture (LP). All subjects will receive ION283. The dose level of 15 mg will be studied in all subjects.

Timeline

Start

2024-12-03

Primary completion

2027-10

Completion

2028-10

Drugs

Indications