Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer (NCT04273243) | Clinical Trial Compass
Active — Not RecruitingNot Applicable
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
United States10 participantsStarted 2020-01-24
Plain-language 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.
Who can participate
Age range12 Months
SexALL
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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
What they're measuring
1
Long-term safety assessment based on Adverse Events (AEs)