Neuronal ceroid lipofuscinosis 2
MONDO:0008769A condition associated with mutation(s) in the TPP1 gene, encoding tripeptidyl-peptidase- 1. The condition is one of a group of genetically heterogeneous neurodegenerative disorders, characterized by accumulation of intracellular lipopigments.
Also known as: late infantile neuronal ceroid lipofuscinosis, CLN2, TPP1 neuronal ceroid lipofuscinosis, ceroid lipofuscinosis, neuronal, type 2, neuronal ceroid lipofuscinosis caused by mutation in TPP1, neuronal ceroid lipofuscinosis type 2, CLN2 disease, CLN2 disease, juvenile (subtype)
9 clinical trials for this condition and its sub-types.
Follow this condition — get notified about new trialsSub-types
Broader categories
-
Gene therapy shot aims to save sight in batten disease kids
Disease control Recruiting nowThis early study tests a one-time gene therapy injection under the retina for children with CLN2 Batten disease who already receive enzyme therapy. The main goal is to check safety in 16 participants, with a secondary look at whether it can preserve vision. It is a first-in-human…
Phase: PHASE1, PHASE2 • Sponsor: Tern Therapeutics, LLC • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC
-
Scientists launch major study to unlock secrets of deadly batten disease
Knowledge-focused Recruiting nowThis study follows 300 people with CLN3 Batten disease, a rare genetic disorder that causes vision loss, seizures, and decline in thinking and movement. Researchers collect samples like blood and spinal fluid to find biological markers that could be used in future treatment trial…
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:08 UTC
-
Scientists track batten disease to unlock its secrets
Knowledge-focused Recruiting nowThis study follows 500 children and adults with Batten disease to learn how the condition naturally changes over time. Researchers will measure movement, thinking, behavior, and daily function using a special rating scale. The goal is to better understand the disease and improve …
Sponsor: University of Rochester • Aim: Knowledge-focused
Last updated Jun 26, 2026 15:36 UTC