Neuronal ceroid lipofuscinosis – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Sat, 20 Jun 2026 04:04:07 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Neuronal ceroid lipofuscinosis – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 First study of TTX-381 gene therapy safety in patients with eye problems caused by CLN2 disease (Neuronal Ceroid Lipofuscinosis Type 2) https://clinicaltrials.eu/trial/first-study-of-ttx-381-gene-therapy-safety-in-patients-with-eye-problems-caused-by-cln2-disease-neuronal-ceroid-lipofuscinosis-type-2/ Wed, 29 Apr 2026 15:06:52 +0000 https://clinicaltrials.eu/trial/first-study-of-ttx-381-gene-therapy-safety-in-patients-with-eye-problems-caused-by-cln2-disease-neuronal-ceroid-lipofuscinosis-type-2/ This clinical trial focuses on evaluating a gene therapy called TTX-381 for patients with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2), specifically targeting eye-related symptoms. CLN2 is a rare genetic condition that affects the nervous system and causes vision problems. The study aims to test how safe and well-tolerated this new treatment is when given to patients.

The treatment involves a special type of medicine called gene therapy that is injected under the retina (the light-sensitive layer at the back of the eye). The medication, TTX-381, uses a harmless virus called AAV9 to deliver a working copy of the CLN2 gene to the cells in the eye. This treatment is designed to help produce a protein that is missing in patients with CLN2 disease.

During the study, patients will receive the treatment and be monitored for about one year to check for any side effects and changes in their vision. The study will look at various aspects of eye health, including measuring the thickness of different layers in the retina using special eye imaging. Doctors will also collect samples to check how the treatment is working in the body.

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