Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

NCT01161576 · clinicaltrials.gov ↗

PHASE1

Phase

COMPLETED

Status

12

Enrollment

OTHER

Sponsor class

Conditions

Batten Disease
Late-Infantile Neuronal Ceroid Lipofuscinosis

Interventions

BIOLOGICAL: AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies
BIOLOGICAL: AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies

Sponsor

Weill Medical College of Cornell University

Collaborators

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