An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
This ongoing study is for children with a bone growth condition called achondroplasia. It's designed to continue looking at a medicine called BMN 111, which was also studied in a previous trial. Children who took part in the earlier study (either BMN 111 or a placebo) might be able to join this one. The main goals are to understand how safe BMN 111 is and if it helps children with achondroplasia grow over a longer period, possibly until they reach their adult height. The medicine is given as a needle jab under the skin once a day. This is a "Phase 2" study, meaning researchers are still gathering more information about the medicine.
Em resumo
Sobre o que é este estudo?
This study is an important next step for children with a genetic condition called achondroplasia. Achondroplasia affects how bones grow, leading to shorter arms and legs. Researchers are testing a medicine named BMN 111, which previous studies have suggested might help with bone growth.
This specific study is an 'extension' of an earlier trial. This means that children who participated in the first study, whether they received BMN 111 or a placebo (a dummy treatment), may now have the chance to continue with BMN 111. The main idea is to see if BMN 111 is safe to use over a longer time and if it continues to help children grow taller until they've reached their full adult height.
By taking part, around 70 children will receive daily doses of BMN 111. The information gathered will help doctors and scientists understand more about how this medicine works and whether it could become a treatment option in the future. It's a way of continuing to learn about the medicine's long-term effects on growth and overall health.
Pontos principais
- This is an extension study for children with achondroplasia.
- It tests an experimental medicine called BMN 111 given daily by injection.
- The study aims to check the long-term safety and effectiveness of BMN 111.
- Participants must have completed a previous BMN 111 study.
- The study will continue until children reach near-adult height.
- Regular health checks and growth monitoring will be part of taking part.
Quem pode ser elegível?
To join this study, children must have already completed a previous study (called 111-206) where they received either the study medicine BMN 111 or a placebo.
Parents or guardians need to be willing to give their written permission for their child to take part. If the child is old enough to understand, they will also be asked to agree in writing. It's also important that both the child and their family are able to follow all the study instructions and attend appointments.
However, some reasons might mean a child can't join. This includes if they stopped the previous study early, if they have certain heart problems, or if they're already taking other specific medications that might interfere with the study medicine. Pregnant individuals or those planning to become pregnant cannot participate. The study team will review all health information to make sure it's safe for every child to take part.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- Did my child complete the previous BMN 111 study (111-206)?
- Are we able and willing to follow all study instructions and attend appointments?
- Is my child currently taking any blood pressure medicines or other specific drugs?
- Does my child have any serious heart conditions?
- Are we planning for pregnancy during the study period?
O que envolve a participação?
If a child joins this study, they will receive BMN 111 as a daily injection under the skin. The dose will depend on their weight and age, as decided during the previous study. The study lasts until each child reaches their estimated adult height, which means it could be several years.
During this time, there will be regular visits to the clinic where doctors will check their health, measure their growth, and possibly take blood or urine samples to see how the medicine is working and to monitor for any side effects. These check-ups are important to make sure the medicine is safe and effective. The exact number and frequency of visits will be explained by the study team, but they will be spread out over the long term.
Riscos e benefícios potenciais
Localizações (16)
- Children's Hospital & Research Center OaklandCódigo postal verificadoOakland, Estados Unidos
- Harbor - UCLA Medical CenterCódigo postal verificadoTorrance, Estados Unidos
- Alfred I. duPont Hospital for ChildrenCódigo postal verificadoWilmington, Estados Unidos
- Universidade EmoryCódigo postal verificadoDecatur, Estados Unidos
- Ann Robert and H. Lurie Children's Hospital of ChicagoCódigo postal verificadoChicago, Estados Unidos
- Cincinnati Childrens HospitalCódigo postal verificadoCincinnati, Estados Unidos
- Vanderbilt University Medical CenterCódigo postal verificadoNashville, Estados Unidos
- Baylor College of MedicineCódigo postal verificadoHouston, Estados Unidos
- Medical College of Wisconsin, Children's HospitalCódigo postal verificadoMilwaukee, United States
- The Children's Hospital at WestmeadCódigo postal verificadoWestmead, Australia
- Murdoch Children's Research InstituteCódigo postal verificadoParkville, Austrália
- Osaka UniversityCódigo postal verificadoOsaka, Japan
Perguntas frequentes
What is achondroplasia?
Achondroplasia is a genetic condition that affects how bones grow, leading to shorter arms and legs.
What is BMN 111?
BMN 111 is an experimental medicine being tested to see if it can help improve bone growth in children with achondroplasia.
Como o medicamento é administrado?
BMN 111 is given as a daily injection (a small jab) under the skin.
Quanto tempo durará o estudo?
The study will last for many years, until your child reaches their full adult height.
Can my child stop taking part if they want to?
Yes, you and your child can decide to leave the study at any time, for any reason.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
Discussão
Discussão da comunidade
Desenvolvido pelo nosso fórum em community.patient.info. Por favor, seja respeitoso — isto não é um conselho médico.