A Phase 1/2 Open Label Study to Evaluate the Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease
Berge Minassian
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.
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
Eligibility
- Age range
- 10–18 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: General Inclusion Criteria * Must give written informed consent (and assent if indicated by patient's age and in accordance with local requirements) and be willing/able to comply with all study requirements. * Aged 10-18 (inclusive) years old at the time of informed consent. * Non-pregnant and non-lactating females * All male participants and women of childbearing potential must refrain from sperm/egg donation from the time of signing the informed consent/assent form until at least 12 weeks (approximately 5 half-lives of ION283) after the dose of Study Drug. * For partici…
Interventions
- GeneticION283
Anti-sense Oligonucleotide therapy that includes intrathecal (IT) injections by lumbar puncture (LP) with dose level of 15 mg.
Location
- Childrens HealthDallas, Texas