A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to <18 Years of Age with Achondroplasia: PROPEL 3

This research study, called PROPEL 3, is investigating a new medicine called Infigratinib for children and young people between 3 and 18 years old who have achondroplasia. Achondroplasia is a genetic condition that affects how bones grow, leading to shorter limbs and sometimes other health challenges. The aim of this study is to see if Infigratinib can help children with achondroplasia grow in height, and improve how their bodies are proportioned, as well as looking at physical abilities and thinking skills. This is a "Phase 3" study, which means the medicine has already been tested in smaller groups, and now researchers need to confirm its effectiveness and safety in a larger group of people.

To make sure the results are reliable, the study is set up so that some children will receive the active medicine (Infigratinib), and others will receive a placebo. A placebo is a dummy pill that looks exactly like the real medicine but contains no active ingredients. Neither the children, their families, nor the doctors will know who is getting which, to ensure everyone's observations are unbiased. This is an important way to truly understand if the medicine is working and whether any improvements are due to the treatment itself.

The study will carefully measure many things over 52 weeks (about one year). The main thing they want to see is if children taking Infigratinib grow more in height compared to those taking the placebo. They will also look at how body parts grow in relation to each other, such as arm and leg length, and head size. Beyond growth, researchers are interested in physical abilities and how the children feel generally, including their thinking and memory skills. Safety is paramount, and researchers will be closely monitoring for any unwanted side effects of the medicine.