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.
Auf einen Blick
Worum geht es in dieser Studie?
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.
Wichtige Erkenntnisse
- 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.
Wer könnte berechtigt sein?
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.
Könnte diese Studie für Sie geeignet sein?
Beantworten Sie diese kurzen Fragen, um zu sehen, ob Sie möglicherweise berechtigt sind. Dies ist nur ein Leitfaden – das Forschungsteam trifft die endgültige Entscheidung.
- 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?
Was beinhaltet die Teilnahme?
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.
Potenzielle Risiken und Vorteile
Standorte (16)
- Children's Hospital & Research Center OaklandVerifizierte PostleitzahlOakland, Vereinigte Staaten
- Harbor - UCLA Medical CenterVerifizierte PostleitzahlTorrance, Vereinigte Staaten
- Alfred I. duPont Hospital for ChildrenVerifizierte PostleitzahlWilmington, Vereinigte Staaten
- Emory UniversityVerifizierte PostleitzahlDecatur, United States
- Ann Robert and H. Lurie Children's Hospital of ChicagoVerifizierte PostleitzahlChicago, Vereinigte Staaten
- Cincinnati Childrens HospitalVerifizierte PostleitzahlCincinnati, Vereinigte Staaten
- Vanderbilt UniversitätsklinikumVerifizierte PostleitzahlNashville, Vereinigte Staaten
- Baylor College of MedicineVerifizierte PostleitzahlHouston, Vereinigte Staaten
- Medical College of Wisconsin, Children's HospitalVerifizierte PostleitzahlMilwaukee, Vereinigte Staaten
- The Children's Hospital at WestmeadVerifizierte PostleitzahlWestmead, Australia
- Murdoch KinderforschungsinstitutVerifizierte PostleitzahlParkville, Australien
- Osaka UniversityVerifizierte PostleitzahlOsaka, Japan
Häufige Fragen
Was ist Achondroplasie?
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.
Wie wird das Medikament verabreicht?
BMN 111 is given as a daily injection (a small jab) under the skin.
Wie lange wird die Studie dauern?
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.
Wie Sie mehr erfahren können
Sprechen Sie immer mit Ihrem Hausarzt oder Spezialisten, bevor Sie sich entscheiden, an einer Studie teilzunehmen.
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