Study of oral nizubaglustat for Niemann-Pick type C
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests an oral medication called nizubaglustat for people with Niemann-Pick type C disease who cannot take or are not satisfied with the current treatment miglustat. It aims to see if the drug can help with symptoms like coordination and movement problems.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of Niemann-Pick type C disease.
- You are either unable or unwilling to take miglustat, or you are unsatisfied with how well miglustat works for you.
- You are at least 4 years old.
- Your neurological symptoms (like trouble walking or coordination) started between ages 2 and 15.
- Your walking and coordination problems, measured by a doctor, are between 3 and 30 points on a scale.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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