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

Running, not enrolling

Conditions studied: CLN6, Batten Disease

In brief

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.

Key facts

Study ID

NCT04273243

Run by

Emily de los Reyes

People needed

10

Starts

2020-01-24

Expected to finish

2027-12-01

Last updated by the study team

2025-10-02

Who can join

Age: 1 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subject received AT-GTX-501 (scAAV9.CB.CLN6) in the study "Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9."
• Subject completed or prematurely discontinued from the study "Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9."
• Subject has a legally authorized representative who has provided written informed consent and authorization for use and disclosure of personal health information or research-related health information.

You may not qualify if…

• None

Where it is running

• Nationwide Children's Hosptial — Columbus, Ohio, United States

Full record on ClinicalTrials.gov

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