Hope for kids with batten disease: new drug trial launches

NCT ID NCT04637282

First seen May 16, 2026 · Last updated May 16, 2026

Summary

This study tests an experimental drug called PLX-200 in children aged 6 to 18 with mild-to-moderate CLN3 disease (a form of Batten disease). The goal is to see if the drug can slow down the disease and is safe. About 39 participants will receive either PLX-200 or a placebo, and researchers will track changes in motor skills, vision, language, and seizures over 60 weeks.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Conditions

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