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

AT-GTX-501-02

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.

2020-01-24

2024-10-01

2024-10-01

Active (not recruiting)

10

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

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'CASE_ONLY', 'timePerspective': 'PROSPECTIVE'}, 'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'During the study, blood samples will be taken for routine lab tests and future tests for safety, effectiveness, or other research assessments.'}, 'enrollmentInfo': {'count': 10, 'type': 'ACTUAL'}}

2023-02-06