A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
Achondroplasia is a genetic condition that affects how bones grow, causing shorter limbs and distinct facial and spine features. This research study looks into a medicine called BMN 111 to see how safe it is and how well it works over a longer period. The study is an extension trial. This means it is only open to children who have already taken part in an earlier study (Study 111-202) and received daily injections of BMN 111 for two years. Parents or carers give the child a daily injection under the skin at home after receiving full training. Doctors will track the children's growth, bone health, and general wellbeing over time. Because participants have already been on the medicine, researchers hope to learn how it supports children as they continue to grow.
Auf einen Blick
Worum geht es in dieser Studie?
Achondroplasia is the most common cause of dwarfism. In children with this condition, a specific protein in the body sends signals that slow bone growth down too much, particularly in the arms and legs. BMN 111 is an experimental medicine designed to help balance out these signals, encouraging bone growth and potentially reducing some complications linked to the condition.
This trial is a follow-on study, often called an extension study. It is designed for children who have already spent two years receiving BMN 111 in an earlier trial. While the first study looked at short-term effects, this next stage checks what happens when children continue using the medicine over several years.
Finding out how a treatment works over the long term is vital for understanding its true benefits and any delayed side effects. The information gathered here will help doctors and scientists understand whether continuous treatment helps children reach their natural growth potential safely.
Wichtige Erkenntnisse
- Follow-on trial studying the long-term safety and effects of BMN 111 in achondroplasia.
- Only for children who completed 24 months in the previous trial (Study 111-202).
- Requires a daily injection under the skin, given by a parent or carer at home.
- Regular hospital appointments will monitor physical growth and general health.
- Participation is entirely voluntary and you can stop at any time.
Wer könnte berechtigt sein?
This study is only open to children aged 7 and older who have already taken part in the earlier research trial (Study 111-202) and completed 24 months of treatment with BMN 111. Both the child and their parents or legal guardians must agree to continue taking part, and parents must be willing to give the daily injection at home.
Children cannot take part if they stopped taking BMN 111 permanently during the previous trial, or if they are taking other medicines that might interfere with the study. If a participant is of childbearing age, they must have a negative pregnancy test and agree to use effective birth control, as the medicine's effects on pregnancy are not fully known.
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.
- Has your child completed 24 months of BMN 111 in Study 111-202?
- Is your child aged 7 or older?
- Are you comfortable learning to give daily injections at home?
- Can you bring your child to the hospital for scheduled study visits?
- Is your child free from other medical conditions that might clash with the study?
Was beinhaltet die Teilnahme?
Taking part involves receiving one injection under the skin every day. Parents or carers are given full training by the medical team so they can comfortably give the injections at home.
Children will attend regular clinic visits for check-ups. During these visits, the study team will measure height, arm and leg length, check blood pressure, and take blood or urine samples. X-rays or scans may also be done to check bone development. The study lasts for several years to follow the children through their growth stages, and the healthcare team will stay in close contact throughout.
Potenzielle Risiken und Vorteile
Standorte (9)
- Children's Hospital & Research Center OaklandVerifizierte PostleitzahlOakland, Vereinigte Staaten
- Harbor - UCLA Medical CenterVerifizierte PostleitzahlTorrance, Vereinigte Staaten
- Ann and Robert H. Lurie Childrens Hospital of ChicagoVerifizierte PostleitzahlChicago, Vereinigte Staaten
- Johns Hopkins McKusick - Institute of Genetic MedicineVerifizierte PostleitzahlBaltimore, Vereinigte Staaten
- Vanderbilt UniversityVerifizierte PostleitzahlNashville, Vereinigte Staaten
- Baylor College of MedicineVerifizierte PostleitzahlHouston, Vereinigte Staaten
- Murdoch KinderforschungsinstitutVerifizierte PostleitzahlParkville, Australien
- Institut NeckerVerifizierte PostleitzahlParis, Frankreich
- Guys & St. Thomas NHS Foundation Trust Evelina HospitalVerifizierte PostleitzahlLondon, Vereinigtes Königreich
Häufige Fragen
What is BMN 111?
BMN 111 is a medicine designed to work with natural body signals to encourage bone growth in children with achondroplasia.
Can my child join if they were not in the first trial?
No. This study is only open to children who have already completed two years in the previous study, known as 111-202.
Wie wird das Medikament verabreicht?
It is given as a daily injection just under the skin. Parents or carers are fully trained to do this at home.
Will taking part cost anything?
No, the study medicine and all study-related visits and tests are provided free of charge, and travel costs are usually covered.
Can we leave the study early if we change our minds?
Yes, you can withdraw your child from the trial at any point, and your normal healthcare will continue as usual.
Wie Sie mehr erfahren können
Sprechen Sie immer mit Ihrem Hausarzt oder Spezialisten, bevor Sie sich entscheiden, an einer Studie teilzunehmen.
Diskussion
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