Cerebral lipidosis with dementia
MONDO:0020143Also known as: cerebral lipidosis
38 clinical trials for this condition and its sub-types, 0 tagged with Cerebral lipidosis with dementia itself.
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Sub-types of Cerebral lipidosis with dementia
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Sandhoff disease 13 trials
3 sub-types
- Sandhoff disease, adult form 1 trial
- Sandhoff disease, infantile form 0 trials
- Sandhoff disease, juvenile form 0 trials
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Tay-Sachs disease 13 trials
4 sub-types
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Gaucher disease type I 12 trials
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Cerebrotendinous xanthomatosis 6 trials
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8 sub-types
- Juvenile neuronal ceroid lipofuscinosis 1 0 trials
- Juvenile neuronal ceroid lipofuscinosis 10 0 trials
- Juvenile neuronal ceroid lipofuscinosis 2 0 trials
- Juvenile neuronal ceroid lipofuscinosis 3 0 trials
- Juvenile neuronal ceroid lipofuscinosis 5 0 trials
- Juvenile neuronal ceroid lipofuscinosis 6 0 trials
- Neuronal ceroid lipofuscinosis 9 0 trials
- Parkinsonism due to ATP13A2 deficiency 0 trials
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Adult Krabbe disease 0 trials
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Adult neuronal ceroid lipofuscinosis 0 trials
5 sub-types
- Adult neuronal ceroid lipofuscinosis 1 0 trials
- Adult neuronal ceroid lipofuscinosis 5 0 trials
- Ceroid lipofuscinosis, neuronal, 4 (Kufs type) 0 trials
- Neuronal ceroid lipofuscinosis 11 0 trials
- Neuronal ceroid lipofuscinosis 13 0 trials
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2 sub-types
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New oral drug shows promise for kids with gaucher disease
Disease control CompletedThis study tested the safety and effectiveness of eliglustat, an oral medication, in 57 children aged 2 to 17 with Gaucher disease types 1 and 3. Some children also received the standard enzyme therapy imiglucerase. Researchers measured how the drug moves through the body, side e…
Phase 3 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Gene therapy trial aims to fix enzyme defect in gaucher disease
Disease control CompletedThis early-stage trial tested a new gene therapy called FLT201 in 10 adults with Gaucher disease type 1. The therapy uses a harmless virus to deliver a working copy of the gene that produces a missing enzyme. The main goal was to check safety and see if the treatment can boost en…
Phase 1 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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New pill shows promise for rare brain disorders in early trial
Disease control CompletedThis study tested an oral drug called AZ-3102 in 13 people with GM2 gangliosidosis (Tay-Sachs or Sandhoff disease) or Niemann-Pick type C disease. The main goal was to check safety and how the drug moves through the body over 12 weeks. It was a randomized, double-blind, placebo-c…
Phase 2 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC
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Light-Based brain cap could replace radiation scans for kids
Knowledge-focused CompletedThis study tested whether two light-based technologies, fNIRS and DCS, can safely measure brain activity in children with rare neurocognitive disorders like Niemann-Pick disease and Smith-Lemli-Opitz syndrome. 73 participants, including healthy volunteers, wore a cap with lights …
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated Sep 18, 2026 00:00 UTC
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Small study tracks rare disease to pave way for future treatments
Knowledge-focused CompletedThis study followed 10 people with late onset Tay-Sachs disease to see how their symptoms and body chemistry changed over six months. Researchers measured balance, coordination, and brain chemicals. The goal was to gather information that will help design better clinical trials f…
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:09 UTC
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Could an antioxidant help gaucher disease? new study investigates
Knowledge-focused CompletedThis study measured levels of brain chemicals related to oxidative stress and inflammation in people with type 1 Gaucher disease and healthy volunteers. Researchers gave 33 participants with Gaucher disease the antioxidant N-acetylcysteine (NAC) for about 90 days to see if it cha…
Phase 2 • Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC
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Could a single DNA test solve the mystery of rare brain diseases in kids?
Knowledge-focused CompletedThis study looked at whether whole genome sequencing (a complete read of a person's DNA) can help diagnose leukodystrophies, a group of rare brain diseases that are hard to identify. Researchers enrolled 236 children with white matter abnormalities on brain scans but no known gen…
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:56 UTC
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New study maps how rare brain diseases worsen over time in kids
Knowledge-focused CompletedThis study followed 31 children with GM1 or GM2 gangliosidosis (including Tay-Sachs and Sandhoff disease) to carefully measure how their neurological symptoms, like walking and speech, change over time. The goal was to create a clear picture of disease progression to help design …
Sponsor: Azafaros B.V. • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:52 UTC