NCT06609889

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

A Phase 1/2 Open Label Study to Evaluate the Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Berge Minassian · Academic / Other
• Sex: All
• Age: 10 Years – 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

A Phase 1/2 Open Label Study to Evaluate the Safety and Efficacy of Intrathecally Administered ION283 in Patients with Lafora Disease A single cohort will be evaluated in the study: N=10 • Initial dose of 15 mg ION283 intrathecal bolus (ITB) injection every 12 weeks. The study consists of 2 periods: * Screening Period: 4 weeks * Open label Treatment Period: 24 months

Conditions

• Lafora Disease

Interventions

Type	Name	Description
GENETIC	ION283	Anti-sense Oligonucleotide therapy that includes intrathecal (IT) injections by lumbar puncture (LP) with dose level of 15 mg.

Timeline

• Start date: 2024-12-03
• Primary completion: 2027-10-01
• Completion: 2028-10-01
• First posted: 2024-09-24
• Last updated: 2025-12-18

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study