Long-term safety of olipudase alfa in young children with ASMD
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study looks at the long-term safety and how well the body accepts olipudase alfa therapy in children under 2 years old who have a certain type of ASMD. It helps understand if the treatment works safely over time.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have ASMD type A/B or B and be under 2 years old, or have ASMD type A (any age).
- You must weigh at least 2 kilograms (about 4.4 pounds).
- You must have a confirmed diagnosis of ASMD through a blood or genetic test.
- You must not have taken any other investigational drug within 30 days or 5 half-lives before joining the study.
- You or your guardian must sign a consent form agreeing to the study.
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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