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

  • STATUS
    Recruiting
  • End date
    Jan 29, 2035
  • participants needed
    13
  • sponsor
    Amicus Therapeutics
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Updated on 19 February 2024
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gene therapy
ceroid lipofuscinosis
cln6
batten
neuronal ceroid lipofuscinosis

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.

Description

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. The assessments described in this long-term follow-up (LTFU) study (AT GTX 501 02) are performed following and in addition to the initial 2 years of assessments in the treatment study (AT-GTX-501-01). In this LTFU study, subjects complete safety and efficacy assessments throughout the study's 13-year duration. Combining the duration of the initial treatment study and this LTFU study, the overall duration reflects a follow-up period up-to 15 years since gene transfer via AT-GTX-501.

The primary outcome for this study is to assess the long-term safety of AT-GTX-501 in subjects with CLN6 Batten disease.

The secondary outcome measure of this study is to assess the long-term efficacy of AT-GTX-501 in subjects with CLN6 Batten disease.

Details
Condition Neuronal ceroid lipofuscinosis, CLN6
Age 1years - 100years
Treatment AT-GTX-501
Clinical Study IdentifierNCT04273243
SponsorAmicus Therapeutics
Last Modified on19 February 2024

Eligibility

 Yes No Not Sure

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
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