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ACTIVE NOT RECRUITING
NCT04273243
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
Sponsor: Emily de los Reyes
View on ClinicalTrials.gov
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.
Official title: Long-Term Follow-Up of AT-GTX-501 scAAV9 Gene Transfer in Subjects With CLN6 Batten Disease
Key Details
Gender
All
Age Range
12 Months - Any
Study Type
OBSERVATIONAL
Enrollment
10
Start Date
2020-01-24
Completion Date
2027-12
Last Updated
2025-10-02
Healthy Volunteers
No
Conditions
Interventions
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.
Locations (1)
Nationwide Children's Hosptial
Columbus, Ohio, United States