At a glance
ClinicalIndex Comparison RecordPhase 1Completed· 10 enrolled
Drug / intervention
AAV2CUhCLN2 (3x10^12 particle units)biological
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.
Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children With Late Infantile Neuronal Ceroid Lipofuscinosis
In Brief
A Phase 1 clinical trial evaluating AAV2CUhCLN2 (3x10^12 particle units) for Batten Disease and Late Infantile Neuronal Ceroid Lipofuscinosis. Completed, enrolled 10 participants across 1 site.
Detailed Summary
The aim of this study is to treat the signs and symptoms of late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal inherited disease in the brain. This will be accomplished by using delivery of a gene (method called gene transfer) to administer to the brain an experimental drug called AAV2CUhCLN2, a gene transfer vector.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
CountriesUnited States
CollaboratorsNathan's Battle Foundation
Timeline
Phase 1CompletedFinished
200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
Enrollment StartJun 2004
First PostedSep 2005
Primary CompletionJun 2019
TodayJul 2026
First PostedSep 8, 2005
Enrollment StartJun 1, 2004
Primary CompletionJun 1, 2019
TodayJul 2, 2026
Enrollment to primary: 15 yearsPosted 20.8 years ago
Interventions
AAV2CUhCLN2 (3x10^12 particle units)biological
gene transfer; one-time administration via neuro surgery procedure