NIEMANN-PICK DISEASE, TYPE C
Clinical trials for NIEMANN-PICK DISEASE, TYPE C explained in plain language.
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Hope for rare brain disease: new drug trial targets movement and daily life
Disease control Recruiting nowThis study tests a drug called N-acetyl-L-leucine (IB1001) in people aged 4 and older with Niemann-Pick disease type C, a rare genetic disorder that affects movement and thinking. The trial compares the drug to a placebo to see if it improves coordination and quality of life. Aft…
Matched conditions: NIEMANN-PICK DISEASE, TYPE C
Phase: PHASE3 • Sponsor: IntraBio Inc • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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Search for NPC clues could speed future treatments
Knowledge-focused Recruiting nowThis study aims to find biological markers that can track the progression of Niemann-Pick type C (NPC), a rare genetic disorder that damages the nervous system. Researchers will evaluate 900 patients of any age through regular checkups, spinal taps, eye exams, and brain scans. Th…
Matched conditions: NIEMANN-PICK DISEASE, TYPE C
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated Jul 02, 2026 00:00 UTC
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New study aims to crack the code of rare brain disease
Knowledge-focused Recruiting nowThis study is creating the first large database for Niemann-Pick type C (NPC), a rare disease that damages the brain, liver, and spleen. Researchers will collect blood samples and medical records from 100 people with NPC to link their genes with their symptoms. The goal is to und…
Matched conditions: NIEMANN-PICK DISEASE, TYPE C
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC