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Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

NCT02725580 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 13

Last updated 2025-10-24

Study results available
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Summary

This is a phase 1/2, open-label, single dose study to evaluate the safety and efficacy of AT-GTX-501 delivered intrathecally into the lumbar spinal cord region of participants with mild to moderate variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6 disease).

Conditions

  • Variant Late-Infantile Neuronal Ceroid Lipofuscinosis

Interventions

GENETIC

AT-GTX-501

CLN6 Gene delivered by Self-Complementary AAV9

Sponsors & Collaborators

  • Emily de los Reyes

    lead OTHER

Principal Investigators

  • Emily de los Reyes, MD · Nationwide Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-05-31
Primary Completion
2022-10-31
Completion
2024-10-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

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 NCT02725580 on ClinicalTrials.gov