Study of oral nizubaglustat for rare genetic disorders
Treatments studied
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new oral medicine, nizubaglustat (AZ-3102), for children and adults (4+) with late-infantile or juvenile Niemann-Pick type C, GM1, or GM2 gangliosidosis. It aims to see if the drug is safe and helps slow disease progression.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be 4 years old or older
- Have a confirmed diagnosis of late-infantile or juvenile Niemann-Pick type C disease, or GM1 or GM2 gangliosidosis (Tay-Sachs, Sandhoff, or GM2AB variant)
- Have the late-infantile or juvenile form of the disease
- Not have certain other serious health problems (detailed info in the full subprotocol)
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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