An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

This study is for children and young people who have already successfully completed a previous study involving BMN 111. Generally, participants need to be at least 6 years old. Girls aged 10 and over, or who have started their periods, will need to take pregnancy tests throughout the study and use effective contraception if they are sexually active. Everyone involved, including parents or guardians, needs to understand the study and agree to take part.

You won't be able to join this study if you stopped the previous BMN 111 study early, or if doctors find any significant heart problems. Also, if your growth has slowed down a lot recently or your growth plates (the parts of bones that grow) have closed, you wouldn't be able to take part. This study also isn't suitable if you're taking other special research medicines, have certain kidney issues, or are pregnant, breastfeeding, or planning to become pregnant. The study team will make sure it's safe and appropriate for everyone who participates.

If you decide to take part, you'll be continuing to receive the BMN 111 medicine, as you would have in the previous study. The medical team will regularly check your health through various assessments, which might include check-ups, tests, and possibly X-rays to monitor your bone growth. Girls will also need to have pregnancy tests if applicable. You or your child will have regular visits with the study team, who will keep a close eye on how you're doing and if there are any side effects.

The study aims to gather information over a longer period, so you'd be involved for an extended time. Details about the exact number of visits, specific tests, and the total length of your participation would be explained fully by the study team. They will also make sure you understand how to take the medicine and what to do if you have any questions or concerns.