Testing nizubaglustat for Gaucher disease type 3
Treatments studied
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests an experimental drug called nizubaglustat (AZ-3102) for people with Gaucher disease type 3. It aims to see if the drug can improve symptoms like anemia, low platelets, and enlarged spleen, which are common in this condition.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have been diagnosed with Gaucher disease type 3.
- You need to be between the ages of 4 and 30.
- You must have at least two areas of impairment measured by a special scoring tool (mSST).
- You should have Gaucher-related anemia, low platelets, or an enlarged spleen.
- You cannot have had enzyme replacement therapy or substrate reduction therapy in the past 12 months.
- If you are a woman who could become pregnant, you must agree to use birth control.
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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