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.
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Broader categories
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Experimental gene therapy aims to halt rare fatal brain disease in children
Disease control OngoingThis early-stage trial tests a one-time gene therapy called CLN-301 in 7 children aged 3 to 10 with CLN3 Batten disease, a rare genetic disorder that causes progressive loss of vision, movement, and thinking skills. The therapy delivers a working copy of the CLN3 gene directly in…
Phase: PHASE1, PHASE2 • Sponsor: Neela Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 13:06 UTC
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Gene therapy for rare brain disease tracked over years
Disease control OngoingThis study follows 10 people with CLN6 Batten disease who received a single dose of gene therapy (AT-GTX-501) in an earlier trial. Researchers are checking long-term safety and how the disease progresses over time. No new treatment is given in this follow-up.
Sponsor: Emily de los Reyes • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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New study aims to measure how CLN2 disease affects Children's development
Knowledge-focused ENROLLING_BY_INVITATIONThis study looks at how children with CLN2 Batten disease develop over time, including their thinking, language, and motor skills. Researchers will compare children who receive treatment (cerliponase alfa) with those who do not. The goal is to find better ways to measure the dise…
Sponsor: Jessica Scherr • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC