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 an experimental drug called N-acetyl-L-leucine (IB1001) for people aged 4 and older with Niemann-Pick disease type C (NPC), a rare genetic disorder that affects movement and thinking. About 53 participants will receive either the drug or a placebo for a period, t…
Matched conditions: NIEMANN-PICK DISEASE, TYPE C
Phase: PHASE3 • Sponsor: IntraBio Inc • Aim: Disease control
Last updated May 26, 2026 07:27 UTC
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New study aims to unlock mysteries of rare brain disease
Knowledge-focused Recruiting nowThis study is creating the first large database of people with Niemann-Pick type C (NPC), a rare disease that affects the brain and other organs. Researchers will collect blood samples and medical records from 100 participants of any age to look for links between genes and sympto…
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 May 26, 2026 07:23 UTC
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Scientists hunt for clues to track rare brain disease
Knowledge-focused Recruiting nowThis study follows up to 900 people with Niemann-Pick disease type C (NPC), a rare genetic disorder that damages the nervous system. Researchers use blood tests, spinal taps, eye exams, and brain scans every six months to find markers that show how the disease progresses. The goa…
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 May 18, 2026 12:09 UTC