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

Part of paid clinical trials in Dallas, Texas.

Sponsor: Berge Minassian
Study ID: NCT06609889
Phase: PHASE1/PHASE2
Status: Recruiting

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Conditions

Lafora Disease

Eligibility Criteria

Sex: ALL
Age: 10 Years - 18 Years
Healthy Volunteers: Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date: Dec 3, 2024
Status verified: Dec 2025
Primary completion: Oct 31, 2027
Completion: Oct 31, 2028

Study Design

Enrollment: 10 participants (estimated)
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Arms

Experimental: ION283 Arm
Open label evaluation of ION283. All subjects with Lafora disease enrolled in this study will receive ION283.

Primary Outcome Measure

Safety of ION283 as assessed by the number of participants with Treatment related AEs [ Time Frame: 2 years ]

Central Contacts

Kristy Riddle, RN, BSN
214-456-9501
[email protected]
Ben Eckert, BA
[email protected]

Locations (1)

Facility	City	State	ZIP	Site coordinators
Childrens Health	Dallas	Texas	75235	Berge Minassian, MD [email protected] Ben Eckert [email protected]

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By research site

Childrens Health· Dallas, TX