Long-term safety study of olipudase alfa in children under 2 with ASMD
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study looks at the long-term safety and effects of olipudase alfa in children under 2 years old with acid sphingomyelinase deficiency (ASMD), a rare genetic condition. It aims to see how the treatment works over time and if it is safe for young children.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Must be under 2 years old (or any age for ASMD type A).
- Must have ASMD type A/B or B, or type A without age restriction.
- Must weigh at least 2 kilograms (~4.4 pounds).
- Must have a confirmed diagnosis of ASMD via laboratory tests or genetic testing.
- Parent or guardian must sign an informed consent form.
- Can have already started olipudase alfa treatment (first dose only) or be eligible to start.
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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