Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

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.

Layout table for study information
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


Group/Cohort Intervention/treatment
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).
Genetic: AT-GTX-501
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.




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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
During the study, blood samples will be taken for routine lab tests and future tests for safety, effectiveness, or other research assessments.


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   12 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The subject population in this Long-Term Follow Up study consists of subjects with CLN6 Batten disease who previously received AT-GTX-501 in the preceding study (AT-GTX-501-01).
Criteria

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

Information from the National Library of Medicine

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


Contacts
Layout table for location contacts
Contact: Amicus Therapeutics Patient Advocacy 609-662-2000 clinicaltrials@amicusrx.com

Locations
Layout table for location information
United States, Ohio
Nationwide Children's Hosptial Recruiting
Columbus, Ohio, United States, 43205
Contact: Physician Investigator         
Sponsors and Collaborators
Amicus Therapeutics
Investigators
Layout table for investigator information
Study Director: Clinical Research Amicus Therapeutics
Layout table for additonal information
Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT04273243    
Other Study ID Numbers: AT-GTX-501-02
First Posted: February 18, 2020    Key Record Dates
Last Update Posted: February 26, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Amicus Therapeutics:
Neuronal ceroid lipofuscinosis
NCL
Gene Transfer
Additional relevant MeSH terms:
Layout table for MeSH terms
Neuronal Ceroid-Lipofuscinoses
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Lipid Metabolism Disorders
Metabolic Diseases