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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Hope fades: trial of Tay-Sachs drug venglustat terminated early
Disease control Stopped earlyThis Phase 3 trial tested an oral drug called venglustat in 75 adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that cause progressive nerve damage. The drug aimed to lower toxic fat buildup in the brain and slow disease worsening. However…
Phase 3 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Experimental cell therapy targets deadly childhood brain cancer
Disease control Stopped earlyThis early-phase trial tested a new immunotherapy approach for children with DIPG, a rare and aggressive brain stem tumor. After standard radiation and chemotherapy, patients received special vaccines and immune cells designed to attack the tumor. The study was small (11 particip…
Phase 1 • Sponsor: University of Florida • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC
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Experimental drug zavesca tested for rare fatal brain diseases in infants
Disease control Stopped earlyThis phase 3 trial tested the drug miglustat (Zavesca) in 30 infants with Sandhoff or Tay-Sachs diseases, rare genetic disorders that destroy nerve cells. The goal was to see if the drug could reduce hospitalizations, seizures, and feeding problems while improving motor function.…
Phase 3 • Sponsor: Tehran University of Medical Sciences • Aim: Disease control
Last updated Jun 26, 2026 17:51 UTC
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Experimental gene therapy targets Tay-Sachs and sandhoff in kids
Disease control Stopped earlyThis early-stage trial tested a gene therapy called AXO-AAV-GM2 in children with Tay-Sachs or Sandhoff disease, rare and fatal genetic brain disorders. The therapy delivers healthy genes directly into the brain and spinal fluid to try to restore a missing enzyme. The study was te…
Phase 1 • Sponsor: Terence Flotte • Aim: Disease control
Last updated Jun 26, 2026 13:03 UTC