AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis

Completed · Phase 1/Phase 2

Conditions studied: Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease

In brief

The investigators propose to assess the safety and efficacy of a new administration method to deliver a biologic to children with a form of Batten disease using an experimental gene transfer procedure. This gene transfer procedure consists of delivering a good copy of the mutated gene to the nerve cells via a virus. These children are born with genetic changes called mutations that result in the inability of the brain to properly recycle proteins. The recycling failure leads to death of the nerve cells in the brain and progressive loss of brain function. Children with Batten disease are normal at birth but by age 2 to 4 have motor and vision problems which progress rapidly to death at age approximately 10 years old. There are no therapies available to treat the disease. The investigators previous clinical trial used a virus called adeno-associated virus 2 (AAV2) as the gene delivery system. That study showed that viral delivery of the gene was safe and showed small, but significant benefits to the recipient. The investigators currently have an IRB approved protocol which uses a slightly different virus called AAVrh.10 as the gene delivery system. This 3rd protocol proposes to use the same virus AAVrh.10 as the gene delivery system and has expanded the eligibility criteria.

Key facts

Study ID
NCT01414985
Run by
Weill Medical College of Cornell University
People needed
8
Starts
2010-04-15
Expected to finish
2017-02-08
Last updated by the study team
2020-08-31

Who can join

Age: 3 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

Where it is running

Full record on ClinicalTrials.gov

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