At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 13 enrolled
Drug / intervention
AT-GTX-501genetic
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9
In Brief
A Phase 2 clinical trial evaluating AT-GTX-501 for Variant Late-Infantile Neuronal Ceroid Lipofuscinosis. Completed, enrolled 13 participants across 1 site.
Detailed Summary
This is a phase 1/2, open-label, single dose study to evaluate the safety and efficacy of AT-GTX-501 delivered intrathecally into the lumbar spinal cord region of participants with mild to moderate variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6 disease).
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
CountriesUnited States
Collaborators--
Timeline
Phase 2CompletedFinished
20162017201820192020202120222023202420252026
First PostedApr 2016
Enrollment StartMay 2016
Primary CompletionOct 2022
Study CompletionOct 2024
TodayJul 2026
First PostedApr 1, 2016
Enrollment StartMay 1, 2016
Primary CompletionOct 1, 2022
Study CompletionOct 1, 2024
TodayJul 2, 2026
Enrollment to primary: 6.4 yearsPosted 10.3 years ago
Interventions
AT-GTX-501genetic
CLN6 Gene delivered by Self-Complementary AAV9