An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
This research is an extension of a previous study and aims to learn more about a medicine called BMN 111 for children who have Achondroplasia. Achondroplasia is the most common form of dwarfism. Researchers want to see how well BMN 111 works and if it's safe to use over a longer period. This study is for children who have already been part of the earlier BMN 111 trial. It's an important step in understanding if this medicine could help children with Achondroplasia in the future. The study will continue to carefully monitor participants to gather more health information.
Auf einen Blick
Worum geht es in dieser Studie?
This research study is a continuation of an earlier trial and focuses on a medicine called BMN 111 for children with Achondroplasia. Achondroplasia is a condition that affects bone growth, leading to shorter arms and legs. The goal of this study is to further understand if BMN 111 can be a helpful treatment option for these children, specifically looking at how well it works and if it is safe when used over a longer time.
Think of this study as the next chapter in learning about BMN 111. The information we gather will help doctors and researchers understand more about how the medicine might support children with Achondroplasia as they grow. This is really important because it helps us learn more about potential treatments that could improve the lives of those living with the condition.
By taking part, children will continue to be monitored by a team of medical professionals. This careful observation will provide valuable insights into the long-term effects of BMN 111, helping to decide if it could become a widely available treatment in the future. Every step of the way, the safety and well-being of the children involved are the top priority.
Wichtige Erkenntnisse
- This study is a follow-up for children who have already been in a previous BMN 111 trial.
- It aims to understand the long-term safety and effectiveness of BMN 111 for Achondroplasia.
- Participants will receive the study medicine and have regular health check-ups.
- The study provides close medical monitoring for participants.
- Participation helps future children with Achondroplasia by advancing medical knowledge.
- You can withdraw from the study at any time without affecting your normal healthcare.
Wer könnte berechtigt sein?
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.
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.
- I (or my child) have already completed the previous BMN 111 study.
- I am at least 6 years old (if applicable, for the child).
- Girls aged 10+ agree to pregnancy tests and contraception if sexually active.
- I do not have any serious heart problems or very slow growth.
- I am not pregnant or breastfeeding, or planning to become pregnant during the study.
- I am generally healthy and able to attend all study visits.
Was beinhaltet die Teilnahme?
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.
Potenzielle Risiken und Vorteile
Locations (24)
- 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 PostleitzahlAtlanta, Vereinigte Staaten
- Ann and Robert H. Lurie Children's Hospital of ChicagoVerifizierte PostleitzahlChicago, Vereinigte Staaten
- Johns Hopkins UniversityVerifizierte PostleitzahlBaltimore, Vereinigte Staaten
- Universität von MissouriVerifizierte PostleitzahlColumbia, Vereinigte Staaten
- Cincinnati Kinderkrankenhaus Medizinisches ZentrumVerifizierte PostleitzahlCincinnati, Vereinigte Staaten
- Baylor College of MedicineVerifizierte PostleitzahlHouston, Vereinigte Staaten
- Seattle Children's HospitalVerifizierte PostleitzahlSeattle, Vereinigte Staaten
- Medical College of Wisconsin, Children's HospitalVerifizierte PostleitzahlMilwaukee, Vereinigte Staaten
- The Children's Hospital at WestmeadVerifizierte PostleitzahlWestmead, Australia
Häufige Fragen
What is Achondroplasia?
Achondroplasia is a genetic condition that affects bone growth, leading to shorter arms and legs. It's the most common type of dwarfism.
What is BMN 111?
BMN 111 is a medicine being researched as a potential treatment for Achondroplasia. This study is looking into its effects over a longer time.
Warum ist diese Studie wichtig?
This study is important because it helps scientists and doctors understand if BMN 111 is safe and effective for long-term use in children with Achondroplasia, which could lead to it becoming a widely available treatment.
Muss ich bezahlen, um an der Studie teilzunehmen?
No, you will not have to pay to participate in the study. All study-related care and medicine are provided free of charge.
Was passiert, wenn ich meine Meinung ändere und nicht mehr teilnehmen möchte?
You are completely free to leave the study at any time, for any reason, without it affecting your medical care in any way.
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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