Study of a new drug for children with achondroplasia
Part of Bones, joints & muscles, Genetic & congenital clinical trials.
This trial tests a new medicine for children with achondroplasia (a common form of dwarfism). It aims to see if the drug can improve growth. The study is for children aged 2.5 to under 11 years old who have not started puberty and can walk without help.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child has a confirmed diagnosis of achondroplasia with a specific FGFR3 gene mutation.
- Your child is between 2.5 and under 11 years old.
- Your child has not yet started puberty (Tanner stage 1).
- Your child can walk without assistance.
- Your child has not had any prior treatments that affect growth, like growth hormone or other experimental drugs for achondroplasia.
- Your child does not have other conditions that cause short stature or serious heart, bone, or sleep problems.
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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