Neuronal ceroid lipofuscinosis
MONDO:0016295A group of inherited progressive degenerative brain diseases characterized clinically by a decline of mental and other capacities, epilepsy, and vision loss through retinal degeneration, and histopathologically by intracellular accumulation of an autofluorescent material, ceroid lipofuscin, in the neuronal cells in the brain and in the retina.
Also known as: NCL, ceroid lipofuscinoses, neuronal ceroid lipofuscinosis
23 clinical trials for this condition and its sub-types.
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Broader categories
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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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Experimental gene therapy aims to halt rare childhood brain disease
Disease control Not yet recruitingThis early-phase trial tests a gene therapy for children with CLN6 Batten disease, a rare and fatal brain disorder. Twelve children will receive a single injection of the therapy into the fluid around the spinal cord. Researchers will check if it is safe and if it can slow the di…
Phase: PHASE1, PHASE2 • Sponsor: The Charlotte and Gwenyth Gray Foundation • Aim: Disease control
Last updated Jun 27, 2026 12:34 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