Polaryx Therapeutics, Inc.
Clinical trials sponsored by Polaryx Therapeutics, Inc., explained in plain language.
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Can a repurposed drug slow rare childhood brain diseases?
Disease control Not yet recruitingThis 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 pro…
Phase: PHASE2 • Sponsor: Polaryx Therapeutics, Inc. • Aim: Disease control
Last updated Aug 02, 2026 00:00 UTC
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Could a common cholesterol drug slow batten disease?
Disease control Not yet recruitingThis 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…
Phase: PHASE3 • Sponsor: Polaryx Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:30 UTC