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

Key facts

Sponsor

Weill Medical College of Cornell University

Principal Investigator

Ronald G. Crystal, MD, MD

Weill Medical College of Cornell University

Intervention

AAV2CUhCLN2 (3x10^12 particle units)(biological)

Enrollment

10 enrolled

Eligibility

3-18 years · All sexes

Timeline

2004 – 2019

Study locations (1)

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

Collaborators

Nathan's Battle Foundation

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

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