Hope for kids with rare batten disease: new drug trial launches
NCT ID NCT04637282
First seen May 16, 2026 · Last updated May 22, 2026 · Updated 3 times
Summary
This study tests a drug called PLX-200 in 39 children aged 6 to 18 with mild-to-moderate CLN3 disease (a rare genetic brain disorder). The goal is to see if the drug can slow down worsening of motor skills and is safe. Participants will receive either PLX-200 or a placebo for about 60 weeks, and their motor function will be tracked using a special rating scale.
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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.
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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Conditions
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