New hope for rare epilepsy: drug ION283 enters human trials

NCT ID NCT06609889

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a new drug called ION283 in 10 people aged 10 to 18 with Lafora disease, a rare and severe form of epilepsy. The drug is given via a spinal injection to see if it is safe and can help control seizures and other symptoms. All participants will receive the same dose, and the study will monitor side effects and changes in disease severity over two years.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • Childrens Health

    RECRUITING

    Dallas, Texas, 75235, United States

    Contact Email: •••••@•••••

    Contact Email: •••••@•••••