NIEMANN-PICK DISEASE, TYPE C
Clinical trials for NIEMANN-PICK DISEASE, TYPE C explained in plain language.
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Hope for rare disease: phase 3 trial of N-Acetyl-L-Leucine for Niemann-Pick type c
Disease control Recruiting nowThis study tests an investigational drug, N-acetyl-L-leucine, in people aged 4 and older with Niemann-Pick disease type C, a rare genetic disorder that affects movement and thinking. Participants receive either the drug or a placebo for a set period, then may continue on the drug…
Matched conditions: NIEMANN-PICK DISEASE, TYPE C
Phase: PHASE3 • Sponsor: IntraBio Inc • Aim: Disease control
Last updated May 12, 2026 13:43 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 17, 2026 06:45 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 for Niemann-Pick type C (NPC), a rare disease that affects the brain, liver, and spleen. Researchers will collect a one-time blood sample and review medical records from 100 people with NPC of any age. The goal is to link genetic ma…
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 11, 2026 20:39 UTC