National Eye Institute 5UG1EY033286-05

NAC Attack, A Phase-3, Multicenter, Randomized, Placebo-Controlled Trial in Patients with Retinitis Pigmentosa

A multi-year clinical trial funded by the National Eye Institute to evaluate the efficacy of oral N-acetylcysteine in preventing cone cell death and preserving vision in patients with retinitis pigmentosa through a randomized, placebo-controlled design.

Description

This project is a multicenter, randomized, double-masked, placebo-controlled clinical trial designed to test the hypothesis that oxidative damage is a major contributor to cone cell death in retinitis pigmentosa (RP) and that long-term oral N-acetylcysteine will promote cone survival and reduce severe visual disability. It aims to determine if long-term NAC treatment can prevent severe visual disability in RP patients regardless of mutation, and to assess if oxidative damage is a key therapeutic target.

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