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

Plain-language summary

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.

Trial details

Phase

N/A

Status

Active, not recruiting

Sponsor

Emily de los Reyes

Intervention

AT-GTX-501

Start date

1/24/2020

Estimated completion

12/1/2027

Countries

United States

Related genes

Locations

• Columbus, United States

Eligibility criteria

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