Study of a new medicine for children with achondroplasia
Part of Bones, joints & muscles, Genetic & congenital clinical trials.
This study tests an oral medicine (ABSK061) to see if it can safely improve growth in children with achondroplasia. It is a phase 1/2 trial, meaning it is early research to find the right dose and check for benefits.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have a confirmed genetic test showing achondroplasia (a change in the FGFR3 gene).
- Your child must be between 3 and 12 years old to join the main part of the study (some younger children may be considered).
- Your child must have been in a separate natural history observation study for at least 6 months before starting this trial.
- Your child must not have had other bone diseases, growth problems, or conditions like untreated thyroid issues or diabetes that affect growth.
- Your child must not have taken any medicines that affect growth, like growth hormone, in the past year.
- Your child must not have severe complications from achondroplasia, such as serious sleep apnea or hydrocephalus that needs treatment.
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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