Neuronal ceroid lipofuscinosis 3

MONDO:0008767

A 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, 12 tagged with Neuronal ceroid lipofuscinosis 3 itself.

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Where it sits in the disease tree

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Sub-types of Neuronal ceroid lipofuscinosis 3

Juvenile neuronal ceroid lipofuscinosis 3 0 trials
Protracted juvenile neuronal ceroid lipofuscinosis 3 0 trials

Including sub-types (12) Tagged with Neuronal ceroid lipofuscinosis 3 (12)

Trials to join now! 6 Not yet recruiting 2 Not yet finished but already full! 3 Completed 1

Experimental drug miglustat tested for rare batten disease

Disease control Completed

This study tested the drug miglustat in 6 adults aged 17 and older with CLN3 Batten disease, a rare genetic disorder that causes nerve damage. The goal was to see if the drug is safe and to find the best dose over 2 years. Researchers tracked side effects and how the drug moved t…

Phase 1/2 • Sponsor: Beyond Batten Disease Foundation • Aim: Disease control

Last updated Jun 26, 2026 14:56 UTC

Neuronal ceroid lipofuscinosis 3

Where it sits in the disease tree

Experimental drug miglustat tested for rare batten disease