A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia

To join this study, we're looking for children with achondroplasia between the ages of 2 and 11. It's important that their achondroplasia has been confirmed by a genetic test, and they must be able to stand on their own without help. Their parents or guardians must be willing and able to give weekly injections and follow all the study's instructions carefully.

There are also some reasons why a child might not be able to join. For example, if they've taken part in another clinical trial recently (unless it was for specific achondroplasia studies), or if their growth plates (the areas where bones grow) have already closed. Children with certain allergies to the medicines being studied, or other medical conditions that affect growth besides achondroplasia, wouldn't be able to participate. Also, if they're currently taking other medicines that influence growth, they might not be suitable.

If your child joins this study, they will receive two different medicines, navepegritide and lonapegsomatropin. These will be given as separate injections under the skin, once a week. Your child's parent or guardian will be trained on how to give these injections at home. There will be regular visits to the clinic so the study team can check on your child's health, measure their growth, and make sure the treatment is going well. These visits will also involve checking on their general health through blood tests and other assessments. The study will last for a period of time, including a follow-up period after the treatment ends, to monitor your child's progress and safety.