Gene therapy for vision loss in CLN2, tested for safety
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This early-phase trial studies a gene therapy medicine called TTX-381 to see if it is safe and tolerable in people with CLN2 disease that affects the eyes. The study also looks at how well the treatment fits into existing eye enzyme therapy care.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your child) have CLN2 mutations on both copies of the gene (biallelic CLN2 mutations).
- Your blood test shows low TPP1 activity in white blood cells.
- You have CLN2 symptoms affecting development or worsening over time, such as seizures or vision loss—or you have an older sibling with confirmed CLN2.
- You are already receiving the eye enzyme treatment cerliponase alfa through a shot into the brain’s fluid (biweekly ICV ERT).
- Your eye measurements on a special eye scan (SD-OCT) fit a specific “accelerated decline” range based on age, or you already received TTX-381 and may be reconsidered later.
- You can follow the study plan for visits for up to 5 years, including agreeing to required birth control rules if applicable.
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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