This study follows, over the long term, people who received the gene transfer therapy AT-GTX-501 for CLN6 Batten disease. It can enroll people who received AT-GTX-501 in the earlier Phase I/IIa gene transfer trial and either completed or discontinued that study. It is a study without an assigned phase and is currently not recruiting but still ongoing (active, not recruiting).
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * 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. Exclusion Criteria: * None