Can a repurposed drug slow rare childhood brain diseases?
NCT ID NCT07740512
First seen Jul 31, 2026 · Last updated Jul 31, 2026
Summary
This phase 2 trial is testing an oral drug called PLX-200 (gemfibrozil) in children aged 2 to 15 with certain lysosomal storage disorders (LSDs), including CLN2, CLN3, Sandhoff disease, and Krabbe disease. The study aims to see if the drug is safe, tolerable, and may slow the progression of these rare genetic conditions that affect the brain and nervous system. Participants will take the drug twice daily for about two years, and researchers will monitor for side effects and measure changes in developmental and behavioral skills.
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), taken orally twice daily
- What this could lead to
- If successful, PLX-200 could offer a treatment to slow or stabilize the progression of certain lysosomal storage disorders in children, potentially improving quality of life and delaying severe symptoms.
- What could go wrong
- This is an early-phase study with a small number of participants, so results may not be conclusive. The drug may not be effective for all types of LSDs, and there is a risk of side effects, including potential liver or muscle issues associated with gemfibrozil.
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.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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