Achondroplasia

This phase 2 trial is testing an experimental drug called TransCon CNP in children with achondroplasia, a common form of dwarfism. The study aims to see if a once-weekly injection under the skin can safely improve growth, measured by height velocity over a year. Participants will…

This trial is testing whether daily injections of recombinant human growth hormone can improve growth in children with achondroplasia, a genetic condition that causes short stature. The study will include children aged 2 to 10 who have not previously been treated with growth horm…

This study tested a once-weekly injection called TransCon CNP in 84 children aged 2 to 11 with achondroplasia, the most common form of dwarfism. The goal was to see if it safely increases growth rate over a year compared to a placebo. After the first year, all children could rece…

This study tests a weekly injection called TransCon CNP in children aged 2 to 10 with achondroplasia, the most common form of dwarfism. The goal is to see if it can safely improve growth over one year. Half the children receive the drug and half receive a placebo, and all childre…

This study tested a daily oral drug called infigratinib in 114 children aged 3 to 18 with achondroplasia, the most common form of dwarfism. The goal was to see if it could safely increase their growth rate compared to a placebo. The trial is now complete, and results will show wh…

This study tested an oral drug called infigratinib in 84 children aged 3 to 11 with achondroplasia, the most common form of dwarfism. The goal was to see if it could safely increase their growth rate. Children took the drug daily, and researchers measured height changes and side …

This study followed 271 children with achondroplasia, ages 2.5 to 17, to understand their growth, medical issues, and quality of life over time. No medication or treatment was given. The goal was to gather natural history data to help design future clinical trials.