A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia
This study is an ongoing project for children and teenagers with achondroplasia. It's for those who have already completed a previous study looking into a medicine called TransCon CNP. The main goal is to understand how safe TransCon CNP is over a long time and how well it works when given as a weekly injection under the skin. Participants will continue to receive the medicine until their growth plates (in their leg bones) have closed, meaning their bones have finished growing. This means the length of time each person is in the study will be different. There will be regular visits every few months to check on their health and how they are responding to the treatment.
De un vistazo
¿De qué trata este estudio?
This clinical trial is designed for children and teenagers who have achondroplasia, a type of dwarfism. It's a special kind of study because it's for those who have already participated in an earlier trial for a medicine called TransCon CNP. The researchers want to find out more about how this medicine works over a long time. They will look closely at its safety, meaning any side effects it might cause, and its effectiveness, which means how well it helps with the condition.
Achondroplasia affects how bones grow, particularly in the arms and legs. TransCon CNP is a medication being studied to see if it can help improve bone growth. In this study, the medicine is given as a weekly injection under the skin. The study is called an 'open-label extension,' which means everyone involved knows they are receiving the active medicine, and it's an extension because it continues treatment for those already familiar with it.
Participants will continue to receive the TransCon CNP medicine until their bones have finished growing. This is checked by looking at special X-rays of their leg bones. For girls, this usually happens around age 16, and for boys, around age 18. This means that each person's time in the study will be different, depending on their individual growth. Regular visits will be scheduled every 12-14 weeks to monitor their progress and well-being.
Puntos clave
- This study is for children and teens with achondroplasia who were in a previous TransCon CNP trial.
- It aims to check the long-term safety and effects of weekly TransCon CNP injections.
- Participants continue treatment until their bones stop growing (around age 16 for girls, 18 for boys).
- Regular check-ups will monitor health, growth, and any side effects.
- Your parent/guardian must give consent and be able to administer the weekly injections.
- La participación es voluntaria, y puedes retirarte en cualquier momento.
¿Quién puede ser elegible?
This study is specifically for children and teenagers aged 3 to 15 years old who have achondroplasia. Crucially, you must have already taken part in a previous clinical trial for TransCon CNP and completed it successfully. This means the researchers already have some information about how you responded to the treatment.
Your parent or legal guardian must agree in writing for you to take part, and they need to be able to give the weekly injections under the skin and follow the study rules. You should also be in good health, according to the doctors' checks, and not have any other medical conditions that would make it unsafe or difficult to take part.
There are also some reasons why someone might not be able to join. For example, if you are allergic to the medicine or its ingredients, or if you are taking other prescription medicines (apart from TransCon CNP). Female participants who could get pregnant must use effective contraception during and for a period after the study. Your vitamin D levels will also be checked to make sure they are not too low.
¿Podría este estudio ser adecuado para ti?
Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.
- Are you between 3 and 15 years old?
- Do you have achondroplasia?
- Have you already completed a previous TransCon CNP study?
- Are your parents/guardians willing to give weekly injections and follow study rules?
- Are you generally healthy, with no known allergies to the study medicine?
- If you are a girl of childbearing potential, are you using effective contraception?
¿En qué consiste la participación?
If you join this study, you will continue to receive the TransCon CNP medicine as a weekly injection under the skin. Your parent or guardian will be trained on how to give these injections at home. The dose will be 100 micrograms of CNP per kilogram of your body weight each week.
You will have regular check-ups at the clinic, usually every 12 to 14 weeks. These visits will involve various assessments, including physical examinations, blood tests, and X-rays to check your bone growth. The purpose of these tests is to monitor your health, look for any side effects, and see how well the treatment is working.
Your participation will continue until your leg bones (femur and tibia) have stopped growing and the growth plates have closed. This will be confirmed by X-rays. For girls, this is generally around 16 years of age, and for boys, around 18 years of age. If your bones haven't finished growing by these ages, you may continue treatment until they do. The total length of time you are in the study will therefore be different for everyone.
Riesgos y beneficios potenciales
Locations (19)
- Ascendis Pharma Investigational SiteCódigo postal verificadoLittle Rock, Estados Unidos
- Ascendis Investigational SiteCódigo postal verificadoAurora, Estados Unidos
- Ascendis Investigational SiteCódigo postal verificadoWilmington, United States
- Ascendis Investigational SiteCódigo postal verificadoSaint Paul, United States
- Ascendis Pharma Investigational SiteCódigo postal verificadoColumbia Falls, United States
- Ascendis Pharma Investigational SiteCódigo postal verificadoBuffalo, United States
- Ascendis Investigational SiteCódigo postal verificadoHouston, Estados Unidos
- Ascendis Pharma Investigational SiteCódigo postal verificadoMadison, United States
- Ascendis Pharma Investigational SiteCódigo postal verificadoParkville, Australia
- Ascendis Investigational SiteCódigo postal verificadoLinz, Austria
- Ascendis Investigational SiteCódigo postal verificadoMontreal, Canadá
- Ascendis Pharma Investigational SiteCódigo postal verificadoCopenhagen, Denmark
Preguntas comunes
What is achondroplasia?
Achondroplasia is a genetic condition that affects how bones grow, leading to shorter arms and legs, and typically shorter stature.
What is TransCon CNP?
TransCon CNP is a new investigational medicine being studied to see if it can help improve bone growth in people with achondroplasia.
How is the medicine given?
The medicine is given as an injection under the skin once a week. A parent or guardian will learn how to give this at home.
¿Cuánto tiempo estaré en el estudio?
The study length is different for everyone. You will continue treatment until your bones stop growing, usually around 16 for girls and 18 for boys.
What happens at clinic visits?
Clinic visits, typically every 3-4 months, involve health checks, blood tests, and X-rays to monitor your progress and safety.
Cómo obtener más información
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
¿Interesado en participar?
Discusión
Discusión comunitaria
Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.