High dose ambroxol for adults with Sanfilippo syndrome
Treatments studied
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This trial tests a high dose of a medicine called ambroxol in adults with MPS III (Sanfilippo syndrome). It aims to see if the drug is safe and can help symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of MPS III (Sanfilippo syndrome) through genetic testing.
- You must be 18 years or older.
- You must be able to take the study drug (ambroxol) by mouth.
- You must avoid grapefruit, grapefruit juice, and grapefruit products for 3 days before each dose and throughout the study.
- If you are a woman of childbearing age, you must have a negative pregnancy test and 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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