ApproaCH: A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial evaluating Efficacy and Safety of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Children with Achondroplasia followed by an Open Label Extension period
The ApproaCH study is a research project looking into a new medicine called TransCon CNP for children and teenagers who have achondroplasia. Achondroplasia affects how bones grow, leading to shorter stature. This study aims to find out if TransCon CNP helps children grow taller over 52 weeks (one year) compared to a 'dummy' medicine (placebo). The researchers will carefully measure growth and check for any side effects. After the first year, all children in the study will then receive the TransCon CNP medicine. The study is run in different hospitals and no personal information will be shared.
Em resumo
Sobre o que é este estudo?
This study, called ApproaCH, is investigating a new medicine named TransCon CNP for children and teenagers living with achondroplasia. Achondroplasia is a condition that affects how bones develop, which can result in shorter height. The main goal of this particular study is to understand if TransCon CNP can help children grow more over a year compared to how they would grow without it.
To do this fairly, some children will receive the new medicine, while others will receive a 'placebo' – this looks exactly like the medicine but contains no active ingredients. Neither the children, their families, nor the doctors will know who is getting which. This helps researchers see if any changes are truly due to the new medicine or if they might happen anyway. After a year, everyone in the study will then get the actual TransCon CNP medicine.
The study also aims to make sure the medicine is safe and to check for any side effects. By learning more about TransCon CNP, researchers hope to find new ways to support children with achondroplasia. Your participation would help us understand if this medicine could make a positive difference in their growth.
Pontos principais
- The study is testing a new medicine (TransCon CNP) for children and teenagers with achondroplasia.
- It aims to see if the medicine helps with growth over one year.
- Some participants will receive the new medicine, others a dummy medicine (placebo) initially.
- After one year, everyone will receive the active medicine.
- Regular clinic visits are needed to check growth and safety.
- Participation is entirely voluntary, and you can withdraw at any time.
Quem pode ser elegível?
This study is open to children and teenagers of all genders who have achondroplasia. There isn't a specific age limit mentioned, meaning it's likely open to a wide range of young people with this condition.
The research team will need to check a few things to make sure the study is right for your child. They'll look at your child's medical history to ensure their health is suitable for taking part and that they meet all other study requirements.
It's important to have a chat with your doctor or the study team directly to confirm if your child meets all the specific criteria needed to join. They can provide all the details and answer any personal questions you might have.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- My child has been diagnosed with achondroplasia.
- My child is a child or teenager (no specific age range given, so check with the study team for detailed limits).
- My child is able to receive weekly injections under the skin.
- My child is generally healthy enough to participate in a clinical study.
- I am able to attend regular clinic visits for my child.
O que envolve a participação?
If your child joins this study, they would receive either the investigational medicine, TransCon CNP, or a placebo (a dummy medicine) once a week as an injection just under the skin. This would happen for 52 weeks, which is about one year. During this time, your child would have regular visits to the clinic so the study team can carefully measure their growth, check their general health, and look for any changes or side effects.
After this first year, all children in the study will then receive the TransCon CNP medicine as part of an 'open-label' period. This means everyone will know they are getting the active medicine, and the weekly injections and regular check-ups will continue. The study team will explain exactly how many visits are needed, what assessments will be carried out at each visit, and the total expected duration of your child's involvement.
Riscos e benefícios potenciais
Localizações (3)
- —Não verificadoIrlanda
- —Não verificadoEspanha
- —Não verificadoDinamarca
Perguntas frequentes
What is achondroplasia?
Achondroplasia is a genetic condition that affects how bones grow, leading to shorter arms and legs, a normal-sized body, and a larger head. It's the most common cause of dwarfism.
What is TransCon CNP?
TransCon CNP is a new medicine being tested. It's designed to help bone growth in children and teenagers with achondroplasia.
O que é um placebo?
A placebo is a 'dummy' treatment that looks exactly like the real medicine but doesn't contain any active ingredients. It helps researchers compare how well the real medicine works.
Como o medicamento será administrado?
The medicine or placebo will be given once a week as an injection just under the skin.
Can I stop my child's participation at any time?
Yes, you are free to withdraw your child from the study at any point, for any reason, and it will not affect their future medical care.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
Interessado em participar?
Discussão
Discussão da comunidade
Desenvolvido pelo nosso fórum em community.patient.info. Por favor, seja respeitoso — isto não é um conselho médico.