Gene therapy for vision loss in CLN2 disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests a new gene therapy, TTX-381, given as an eye injection to slow or stop vision loss in children with CLN2 disease. It is for children who are already receiving enzyme replacement therapy and are in a stage of rapid vision decline.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have a confirmed CLN2 diagnosis with two mutations in the CLN2 gene and low TPP1 enzyme activity.
- Your child must be between 2 and 7 years old at the start of the study.
- Your child must be currently receiving enzyme replacement therapy (cerliponase alfa) through a port in the brain every two weeks.
- Your child must have vision loss that is getting worse, with specific retinal thickness measurements between 140 and 210 micrometers in both eyes.
- Your child must not have had gene therapy or a bone marrow transplant before, and must not have had more than 3 eye injections of cerliponase alfa.
- Your child must be able to attend study visits for 5 years.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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