A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
This important study is looking for babies and toddlers (up to two years old) with achondroplasia to test a new medicine called Navepegritide (also known as TransCon CNP). Achondroplasia is a condition that affects how bones grow. For one year, some children will receive the new medicine through a weekly injection, while others will get a placebo, which is a dummy medicine with no active ingredients. The main goal is to find out if Navepegritide is safe and if it helps children grow better than those who receive the placebo. Doctors will carefully monitor the children throughout the study to understand the medicine's effects.
De un vistazo
¿De qué trata este estudio?
This study is about a new medicine called Navepegritide (sometimes called TransCon CNP) for very young children, specifically babies and toddlers up to two years old, who have a condition called achondroplasia. Achondroplasia is a genetic condition that affects how bones grow, leading to shorter limbs. The researchers want to see if this new medicine can help improve growth and if it is safe for these young children.
To do this, some children in the study will receive the new medicine, while others will receive a placebo. A placebo looks just like the real medicine but contains no active ingredients; it helps researchers fairly compare the effects of the new medicine. Neither the families nor the doctors will know who is getting which treatment until the study is over. This is called a 'double-blind' study and helps ensure the results are as fair and unbiased as possible. The medicine or placebo will be given as an injection under the skin once a week for a year.
The study is in 'Phase 2,' which means it's one of the earlier stages of testing. This phase focuses on checking the safety of the medicine and getting an idea of how well it works. The information gathered from this study will be crucial in deciding if the medicine should be tested in more children in the future.
Puntos clave
- This study tests a new medicine for babies and toddlers with achondroplasia.
- It aims to check the medicine's safety and how it affects growth.
- Children will receive weekly injections of either the medicine or a placebo for one year.
- Regular clinic visits and health checks are part of the study.
- La participación es voluntaria, y puedes retirarte en cualquier momento.
- The study is in an earlier stage of research (Phase 2).
¿Quién puede ser elegible?
To join this study, your child must have achondroplasia, which has been confirmed by a genetic test. They need to be under two years old when they start the study. Also, you as their parent or guardian must be willing to give regular injections at home and attend all necessary appointments.
There are some reasons why a child might not be able to join. For example, if they were born very prematurely, or if they have a severe allergic reaction to certain ingredients in the medicine. Children whose achondroplasia is due to a specific genetic type called 'homozygous' will also not be able to participate. Also, if a child is expected to need surgery during the study period, they generally won't be able to join.
The study team will check your child's medical history, conduct examinations, and do some tests to make sure it's safe for them to take part and that they meet all the study requirements.
¿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.
- Is your child under 2 years old?
- Does your child have achondroplasia confirmed by a genetic test?
- Are you able and willing to administer weekly injections?
- Are you able to attend regular clinic appointments?
- Does your child not have a severe allergy to the study medicine's ingredients?
¿En qué consiste la participación?
If your child joins this study, they will receive either the study medicine (Navepegritide) or a placebo, given as an injection under the skin once a week for 52 weeks (one year). You, as the parent or caregiver, will be taught how to give these injections at home. Throughout the year, your child will need to have regular check-ups at the study clinic. These visits will involve physical examinations, taking measurements of your child's growth, and some blood tests to check their health and how the medicine is affecting them. There will also be some scans, like X-rays, to look at their bones. After the year of treatment, there will be follow-up visits to continue monitoring your child's health and development. The total duration of active treatment is one year, with follow-up appointments extending beyond that.
Riesgos y beneficios potenciales
Locations (18)
- Ascendis Investigational SiteCódigo postal verificadoSaint Paul, United States· Reclutamiento
- Ascendis Investigational SiteCódigo postal verificadoHouston, Estados Unidos· Reclutamiento
- Ascendis Investigational SiteCódigo postal verificadoMadison, United States· Reclutamiento
- Ascendis Investigational SiteCódigo postal verificadoParkville, Australia· Reclutamiento
- Ascendis Investigational SiteCódigo postal verificadoLinz, Austria· Reclutamiento
- Ascendis Investigational SiteCódigo postal verificadoMontreal, Canadá· Reclutamiento
- Ascendis Investigational SiteCódigo postal verificadoCopenhagen, Denmark· Reclutamiento
- Ascendis Investigational SiteCódigo postal verificadoHelsinki, Finland· Reclutamiento
- Ascendis Investigational SiteCódigo postal verificadoParís, Francia· Reclutamiento
- Ascendis Investigational SiteCódigo postal verificadoBerlín, Alemania· Reclutamiento
- Ascendis Investigational SiteCódigo postal verificadoCologne, Germany· Reclutamiento
- Ascendis Investigational SiteCódigo postal verificadoDublin, Ireland· Reclutamiento
Preguntas comunes
What is achondroplasia?
Achondroplasia is a genetic condition that affects how bones grow, particularly in the arms and legs, leading to a shorter stature.
¿Qué es un placebo?
A placebo is a 'dummy' medicine that looks like the real treatment but contains no active ingredients. It helps researchers fairly compare the effects of the new medicine.
Will my child definitely get the new medicine?
No, there's a 2 in 3 chance your child will receive the new medicine and a 1 in 3 chance they will receive the placebo. Neither you nor the doctors will know which until the study ends.
¿Cuánto tiempo estará mi hijo en el estudio?
Your child will receive the study treatment (or placebo) for one year, with additional follow-up visits after that period.
Can I stop my child's participation at any time?
Yes, you have the right to withdraw your child from the study at any point, and it will not affect their future medical care.
Cómo obtener más información
Ascendis Registry Inquiries
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.