Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
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|ClinicalTrials.gov Identifier: NCT04273243|
Recruitment Status : Active, not recruiting
First Posted : February 18, 2020
Last Update Posted : December 20, 2021
|Condition or disease||Intervention/treatment|
|CLN6 Batten Disease||Genetic: AT-GTX-501|
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.
|Study Type :||Observational|
|Actual Enrollment :||10 participants|
|Official Title:||Long-Term Follow-Up of AT-GTX-501 scAAV9 Gene Transfer in Subjects With CLN6 Batten Disease|
|Actual Study Start Date :||January 24, 2020|
|Estimated Primary Completion Date :||January 2035|
|Estimated Study Completion Date :||January 2035|
Subjects who received AT-GTX-501 gene transfer
Subjects with CLN6 Batten disease who previously received AT-GTX-501 in the preceding study (Study AT-GTX-501-01).
No study drug is administered in this study. Subjects who received AT-GTX-501 in a previous trial will be evaluated in this trial for long-term safety and efficacy.
- Long-term safety assessment based on Adverse Events (AEs) [ Time Frame: up to 13 years ]All AEs that occur during this study will be classified as treatment-emergent adverse events (TEAEs), as AT-GTX-501 was previously received by all subjects in this study.
- Hamburg Scale [ Time Frame: up to 13 years ]The Hamburg scale is an established tool to capture the rate of decline or regression. From the Hamburg Scale, the individual motor and language scores and the motor plus language aggregated score will be summarized.
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04273243
|United States, Ohio|
|Nationwide Children's Hosptial|
|Columbus, Ohio, United States, 43205|
|Study Director:||Clinical Research||Amicus Therapeutics|