First Human Study to Test Gene Therapy TTX-381 for Eye Problems in Children with CLN2 Batten Disease Evaluating Safety and Tolerability with Dose Escalation

What this Trial Is About

Researchers are evaluating TTX-381, a gene therapy, in a first-in-human, open-label study to treat eye problems caused by Neuronal Ceroid Lipofuscinosis Type 2 CLN2, also known as Batten disease. This condition results from a faulty gene causing lack of an important enzyme, leading to damage in the brain, spinal cord, and eyes, affecting vision and other functions. The study aims to understand the safety and tolerability of TTX-381 to address these ocular issues in affected children. Participants will receive a single dose of TTX-381 injected under the retina of one eye, while the other eye will serve as a control for comparison. The study includes several dose levels, such as 21010 and 61010 genome copies per eye, with an expansion group receiving the lower dose as determined by an independent committee. This dose-escalation design helps researchers monitor effects at different doses and assess safety. During the five-year follow-up, participants will have regular assessments including eye scans to measure retinal thickness and damage areas, testing for the gene therapy product in eye fluid, and monitoring for any side effects or adverse events. The study team will also collect urine and tear samples to evaluate shedding of the therapy. Safety and eye health will be closely observed throughout the study to gather comprehensive data on the treatments impact.

A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease