Niemann-Pick type C disease
Clinical trials for Niemann-Pick type C disease explained in plain language.
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New pill hopes to slow rare brain disease in kids
Disease control Recruiting nowThis study tests an oral drug called nizubaglustat in children and teens aged 4 and older with Niemann-Pick type C disease, a rare genetic disorder that affects movement and thinking. Participants will take the drug or a placebo for 18 months to see if it slows the disease and im…
Matched conditions: NIEMANN-PICK TYPE C DISEASE
Phase 3 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Sep 20, 2026 00:00 UTC
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New pill hopes to tame rare childhood brain diseases
Disease control Recruiting nowThis Phase 3 trial tests an oral drug called nizubaglustat (AZ-3102) in people aged 4 and older with Niemann-Pick type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. The study lasts 18 months and compares the drug to a placebo to see if it is safe and can slow disease prog…
Matched conditions: NIEMANN-PICK TYPE C DISEASE
Phase 3 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Sep 18, 2026 00:00 UTC
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New hope for rare brain diseases: long-term drug trial now recruiting
Disease control Recruiting nowThis study tests the long-term safety and effectiveness of a daily medication called nizubaglustat in people with two rare genetic diseases that affect the brain and body. About 21 participants who were in a previous study or who have been on a similar drug (miglustat) will take …
Matched conditions: NIEMANN-PICK TYPE C DISEASE
Phase 2 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC