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Study identifier: NCT06609889 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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

Condition: Lafora Disease  ·  Sponsor: Berge Minassian

PhasePhase 1/Phase 2
Planned participants10
Who can joinAll sexes, 10 Years to 18 Years
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Kristy Riddle, RN, BSN  ·  214-456-9501  ·  LDRecruiting@utsouthwestern.edu

Ben Eckert, BA  ·  LDRecruiting@utsouthwestern.edu

Always discuss trial participation with your own doctor first.

Locations (1)

Childrens HealthDallas, Texas, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT06609889