Status:
ACTIVE_NOT_RECRUITING
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
Lead Sponsor:
Emily de los Reyes
Conditions:
CLN6
Batten Disease
Eligibility:
All Genders
12+ years
Brief 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.
Detailed Description
This is a long-term safety and efficacy study in subjects with CLN6 Batten disease (also know as variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene \[vL...
Eligibility Criteria
Inclusion
- 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
- None
Key Trial Info
Start Date :
January 24 2020
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 1 2027
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT04273243
Start Date
January 24 2020
End Date
December 1 2027
Last Update
October 2 2025
Active Locations (1)
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1
Nationwide Children's Hosptial
Columbus, Ohio, United States, 43205