https://clinicaltrials.gov/ct2/show/NCT04273243
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04273243
Recruitment Status : Recruiting
First Posted : February 18, 2020
Last Update Posted : February 26, 2020
See Contacts and Locations
Sponsor:
Amicus Therapeutics
Information provided by (Responsible Party):
Amicus Therapeutics
Study DetailsTabular ViewNo Results PostedDisclaimerHow to Read a Study Record
Study Description
Go to sections
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.
Condition or disease Intervention/treatment
CLN6
Batten Disease
Genetic: AT-GTX-501
Detailed 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.
Study Design
Go to sections
Study Type : Observational
Estimated Enrollment : 13 participants
Observational Model: Case-Only
Time Perspective: Prospective
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
