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

NCT06609889 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2025-12-18

No results posted yet for this study

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.

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.

Sponsors & Collaborators

  • Elpida Therapeutics SPC

    collaborator INDUSTRY

  • Berge Minassian

    lead OTHER

Principal Investigators

  • Berge Minassian, MD · University of Texas Southwestern Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
10 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-12-03
Primary Completion
2027-10-31
Completion
2028-10-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06609889 on ClinicalTrials.gov