Neuronal ceroid lipofuscinosis 3
MONDO:0008767A condition associated with mutation(s) in the CLN3 gene, encoding battenin. The condition is one of a group of genetically heterogeneous neurodegenerative disorders, characterized by accumulation of intracellular lipopigments.
Also known as: CLN3, CLN3 neuronal ceroid lipofuscinosis, Juvenile CLN3 Disease, ceroid lipofuscinosis, neuronal, type 3, neuronal ceroid lipofuscinosis 3, neuronal ceroid lipofuscinosis caused by mutation in CLN3, neuronal ceroid lipofuscinosis type 3, CLN3 disease
12 clinical trials for this condition and its sub-types.
Follow this condition — get notified about new trialsSub-types
Broader categories
-
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
-
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