Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Hypochondroplasia: ACCEL OLE
This study, called ACCEL OLE, is an ongoing, long-term part of a previous research project. It's designed to understand more about a medicine called infigratinib, which is for children with a condition called hypochondroplasia. Hypochondroplasia affects how bones grow. Researchers want to see if this medicine is safe over a longer period, checking for any side effects and how serious they might be. They will also look at how children grow, including their height and body shape, and how their quality of life might be affected. This helps doctors learn if infigratinib can be a helpful treatment option in the future.
Em resumo
Sobre o que é este estudo?
This research study is looking at a medicine called infigratinib for children who have a condition called hypochondroplasia. Hypochondroplasia is a genetic condition that affects how bones grow, leading to shorter limbs and sometimes other health differences. The medicine, infigratinib, works by targeting specific pathways in the body that are involved in bone growth. This study is an important follow-up to an earlier project, meaning those who participated before are continuing to be observed.
The main goal of this long-term study is to understand the safety of infigratinib over time. Doctors want to know about any side effects that might happen, how often they occur, and how serious they are. They will also keep a close eye on children's overall health through regular checks, including blood tests, eye exams, dental checks, and scans like X-rays to see bone changes. This helps to build a full picture of the medicine's effects.
Beyond safety, the study is also interested in how infigratinib might affect growth. Researchers will measure changes in height and body proportions, like the length of arms and legs. They'll also look at things like body composition (how much muscle and fat a child has) and how it affects their quality of life, using special questionnaires for children and their parents. All this information helps doctors learn more about hypochondroplasia and if infigratinib could be an effective and safe treatment for children in the long run.
Pontos principais
- This is a long-term follow-up study for children with hypochondroplasia already on infigratinib.
- It aims to check the safety and potential benefits of infigratinib over time.
- Researchers will track growth, body changes, and overall health.
- The study also looks at how the medicine affects a child's quality of life.
- Regular health checks, including scans and blood tests, are part of participation.
Quem pode ser elegível?
This study is open to children who have hypochondroplasia. It's specifically for those who have already taken part in the earlier phases of this research and have been taking the study medicine, infigratinib.
Because it's an 'extension' study, it means participants have already met specific health requirements from the previous study phase. This helps ensure that the medicine has been tolerated well so far and allows for continued monitoring of its long-term effects.
If your child has hypochondroplasia and has previously been involved in an infigratinib study, you might be eligible to continue in this particular long-term follow-up. Both boys and girls can take part.
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.
- Does my child have hypochondroplasia?
- Has my child previously taken part in a study for infigratinib?
- Is my child currently taking infigratinib as part of a clinical trial?
- Am I open to my child having regular follow-up appointments and tests?
O que envolve a participação?
If you or your child decides to take part in this study, it means you'll continue to receive the study medicine, infigratinib. You'll have regular visits to the clinic, where the study team will carry out various checks and assessments. These will include looking at any possible side effects, checking your child's height and other body measurements, and having routine health checks like blood tests and vital signs. There will also be special checks such as eye and dental exams, as well as X-rays and DXA scans to monitor bone health.
Participants will also complete questionnaires about their quality of life, and parents might have interviews to discuss the benefits of the treatment. The total duration of participation will depend on the ongoing nature of this long-term study, as it's designed to gather information over an extended period.
Riscos e benefícios potenciais
Localizações (2)
- —Não verificadoEspanha
- —Não verificadoNoruega
Perguntas frequentes
What is hypochondroplasia?
Hypochondroplasia is a condition that affects bone growth, leading to shorter arms and legs, and sometimes a shorter stature overall.
What is infigratinib?
Infigratinib is a medicine being studied to see if it can help with bone growth in children with hypochondroplasia.
Why is this called an 'extension' study?
It's an extension study because it's a continuation for children who have already been taking part in an earlier study of infigratinib, to see its long-term effects.
What kind of tests will be done?
Tests include checking for side effects, measuring height and body parts, blood tests, eye and dental checks, and X-rays to look at bones.
Will this study guarantee a cure for hypochondroplasia?
No, this study is to learn more about the medicine and its effects; it doesn't guarantee a cure or specific outcome, but it helps doctors understand potential treatments.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
Discussão
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