NCT04273243

Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer

Long-Term Follow-Up of AT-GTX-501 scAAV9 Gene Transfer in Subjects With CLN6 Batten Disease

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 10 (actual)
• Sponsor: Emily de los Reyes · Academic / Other
• Sex: All
• Age: 12 Months
• Healthy volunteers: Not accepted

Summary

This is a long-term safety and efficacy study in subjects with CLN6 Batten disease who previously received a single intrathecal administration of AT-GTX-501.

Detailed description

This is a long-term safety and efficacy study in subjects with CLN6 Batten disease (also know as variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene \[vLINCL6\] disease),who previously received a single intrathecal administration of AT-GTX-501. The assessments described in this long-term follow-up (LTFU) study (AT-GTX-501-02) are performed following and in addition to the initial 2 years of post-treatment assessments in the treatment study (AT-GTX-501-01). In this LTFU study, subjects complete safety and efficacy assessments throughout the study's 3-year duration. Combining the duration of the initial treatment study and this LTFU study, the overall duration reflects a follow-up period up-to 5 years since gene transfer via AT-GTX-501. The primary outcome for this study is to assess the long-term safety of AT-GTX-501 in subjects with CLN6 Batten disease. The secondary outcome measure of this study is to assess the long-term efficacy of AT-GTX-501 in subjects with CLN6 Batten disease.

Conditions

• CLN6
• Batten Disease

Interventions

Type	Name	Description
GENETIC	AT-GTX-501	No study drug is administered in this study. Subjects who received AT-GTX-501 in a previous trial will be evaluated in this trial for long-term safety and efficacy.

Timeline

• Start date: 2020-01-24
• Primary completion: 2026-12-01
• Completion: 2027-12-01
• First posted: 2020-02-18
• Last updated: 2025-10-02

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study