NCT06609889View on ClinicalTrials.gov

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

PHASE1/PHASE2RecruitingINTERVENTIONAL

Enrollment

10

Participants

Timeline

Start Date

December 3, 2024

Primary Completion Date

October 31, 2027

Study Completion Date

October 31, 2028

Conditions

Lafora Disease

Interventions

GENETIC

ION283

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

Trial Locations (1)

75235

RECRUITING

Childrens Health, Dallas

Sponsors

Collaborators (1)

Elpida Therapeutics SPC

All Listed Sponsors

collaborator

Elpida Therapeutics SPC

INDUSTRY

lead

Berge Minassian

OTHER