Study of vosoritide for children with Noonan syndrome and short stature
Treatments studied
Part of Bones, joints & muscles, Genetic & congenital, Heart & circulation, Hormones & metabolism, Skin clinical trials.
This trial tests a new drug, vosoritide, to help children with Noonan syndrome who are still growing but are shorter than expected despite growth hormone treatment. It aims to see if the drug can improve their growth.
Summary written for real people, not researchers, by Clin2.
Who can take part
- The child must be between 3 and 11 years old for girls, or between 3 and 12 years old for boys.
- The child must have a doctor's diagnosis of Noonan syndrome.
- The child's height must be below the 10th percentile for their age and sex.
- The child must be in Tanner stage 1 (prepubertal).
- The child must have been treated with growth hormone before or currently.
- The child's growth rate must be below normal for their age and sex.
- The child must not have other conditions that cause short stature.
- The child's bone age must not be more than 2 years ahead of their actual age.
- The child must not have uncorrected heart defects that increase risk.
- The child must not have had leg-lengthening surgery or plan to have it during the study.
- The child must not have had any bone surgery (other than teeth) or plan to have it during 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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