Gene Therapy for Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease
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|ClinicalTrials.gov Identifier: NCT02725580|
Recruitment Status : Active, not recruiting
First Posted : April 1, 2016
Last Update Posted : April 13, 2020
|Condition or disease||Intervention/treatment||Phase|
|Variant Late-Infantile Neuronal Ceroid Lipofuscinosis||Genetic: AT-GTX-501||Phase 1 Phase 2|
This is an open-label, single-dose study of AT-GTX-501 administered by a single intrathecal injection. Safety and efficacy are evaluated over a 2 year period. The efficacy assessments in this study are to evaluate motor, language, visual, and cognitive function, as well as survival and other outcome measures. Subjects are tested at baseline, receive AT-GTX-501 on Day 0, and return for visits on Days 7, 14, 21, and 30, and then every 3 months until Month 24. Following completion of this study, there is a long-term follow up study in which data will continue to be collected (Study AT-GTX-501-02 / NCT04273243).
For more information about this study, please contact Amicus Therapeutics Patient Advocacy at firstname.lastname@example.org or +1 609-662-2000.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||13 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9|
|Actual Study Start Date :||March 2016|
|Estimated Primary Completion Date :||November 2021|
|Estimated Study Completion Date :||November 2021|
Experimental: Open Label
Subjects with diagnosis of vLINCL6 Batten disease will receive a single intrathecal injection into the lumbar spinal cord region of AT-GTX-501.
CLN6 Gene delivered by Self-Complementary AAV9
Other Name: scAAV9.CB.CLN6
- Safety evaluation based on the development of dose-limiting toxicity (DLT). [ Time Frame: 24 months ]The DLT is defined as any unanticipated AE that is considered related to AT-GTX-501 and is Common Terminology Criteria for Adverse Events Grade 3 or higher.
- Hamburg Scale [ Time Frame: 24 months ]The Hamburg scale is an established tool to capture the rate of decline or regression.
- Unified Batten Disease Rating Scale (UBDRS) [ Time Frame: 24 months ]The UBDRS scale was developed to monitor rate of progression. This scale includes assessment of extrapyramidal movement behavior and seizures.
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): NCT02725580
|United States, Ohio|
|Nationwide Children's Hosptial|
|Columbus, Ohio, United States, 43205|
|Study Director:||Clinical Research||Amicus Therapeutics|