NCT00151216View on ClinicalTrials.gov

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

PHASE1CompletedINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

June 30, 2004

Primary Completion Date

June 30, 2019

Study Completion Date

June 30, 2019

Conditions
Batten DiseaseLate Infantile Neuronal Ceroid Lipofuscinosis
Interventions
BIOLOGICAL

AAV2CUhCLN2 (3x10^12 particle units)

gene transfer; one-time administration via neuro surgery procedure

Trial Locations (1)

10021

New York Presbyterian Hospital - Weill Medical College of Cornell University, New York

All Listed Sponsors
collaborator

Nathan's Battle Foundation

OTHER

lead

Weill Medical College of Cornell University

OTHER

NCT00151216 - Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis | Biotech Hunter | Biotech Hunter