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

Key facts

Sponsor

Weill Medical College of Cornell University

Principal Investigator

Ronald G Crystal, MD, MD

Weill Medical College of Cornell University

Intervention

AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies(biological)

Enrollment

12 enrolled

Eligibility

2-18 years · All sexes

Timeline

2010 – 2020

Study locations (1)

• Weill Cornell Medical College, New York, New York, United States

Collaborators

National Institutes of Health (NIH)

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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