Could a common cholesterol drug slow batten disease?
NCT ID NCT04637282
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 3 trial tests whether PLX-200 (gemfibrozil) can safely slow the progression of CLN3 disease, a rare genetic disorder that causes loss of motor skills, vision, and seizures in children. About 39 kids aged 6-18 will receive either the drug or a placebo for up to 96 weeks. The main goal is to see if motor decline is reduced.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- PLX-200 (gemfibrozil)
- What this could lead to
- If it works, this could slow the loss of motor skills and other functions in children with CLN3 disease, offering a first treatment option.
- What could go wrong
- This is a small, early-stage trial (39 participants) testing a repurposed drug. It may not show significant benefit, and side effects are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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