Phase II study of TYRA-300 in children with achondroplasia
This study is testing a new medicine called dabogratinib (TYRA-300) for children aged 3 to 10 who have achondroplasia and whose bones are still growing. The main goal is to find out if the medicine is safe, how well children tolerate it, and what dose might work best. Some children will start the medicine right away, while others will be observed for a while first to understand their natural growth. All children may have the chance to continue the medicine for a longer time, even up to several years. This research is important to potentially help children with achondroplasia grow more.
En un coup d'œil
De quoi parle cette étude ?
Achondroplasia is a condition that affects how bones grow, leading to shorter-than-average height. This study is testing a new medicine called dabogratinib, also known as TYRA-300, specifically for children with achondroplasia whose growth plates are still open. Growth plates are special areas at the ends of bones that allow children and teenagers to grow taller. By studying these children, researchers hope to see if dabogratinib can help improve bone growth.
The main aims of this study are to check if dabogratinib is safe for children to take and to find the right amount, or 'dose,' that might be most helpful. This is called a Phase II study, which means it's one of the first careful steps to see if a new medicine works and is safe for people, after initial safety tests. The study will involve several groups of children, each helping researchers learn different things about the medicine and its effects.
Ultimately, this research could lead to a new treatment option for children with achondroplasia. It's a long process, but each step helps medical science move closer to finding better ways to help children affected by this condition. The study is carefully designed to gather important information while keeping the children's safety and well-being as the top priority.
Points clés
- Tests a new medicine (dabogratinib) for achondroplasia.
- For children aged 3-10 with open growth plates.
- Aims to find safe and effective dose levels.
- Participation involves regular clinic visits and taking daily medication.
- Children may be in the study for several years.
- Carefully monitors safety and growth.
Qui peut être éligible ?
This study is looking for children with achondroplasia who meet certain criteria. Generally, children aged between 3 and 10 years old can take part. It's important that they have been diagnosed with achondroplasia through genetic testing, specifically showing a gene change called FGFR3 G380R.
Another key requirement is that their 'growth plates' must still be open. These are the parts of the bones that are still developing and allowing growth, which a doctor can check with an X-ray. Children also need to be able to take medicine by mouth, usually a sprinkle capsule or mini-tablet.
There are also some important safety checks. For example, if a child is 10 years or older or has started periods, they will need pregnancy tests. Also, sexually active teenagers and their partners would need to use very effective birth control during the study. It's also important that participants avoid certain fruits like grapefruit and star fruit, as they can interfere with the medicine.
Cet étude pourrait-elle vous convenir ?
Répondez à ces questions rapides pour voir si vous êtes éligible. Ceci est un guide uniquement — l'équipe de recherche prend la décision finale.
- Is your child aged between 3 and 10 years old?
- Does your child have achondroplasia with the FGFR3 G380R gene change?
- Has a doctor confirmed your child's growth plates are still open?
- Can your child take medicine by mouth (sprinkle capsule or mini-tablet)?
- Can your family attend regular appointments and follow study rules?
- Is your child able to stand and walk on their own?
En quoi consiste la participation ?
If your child takes part in this study, they will be given the new medicine, dabogratinib (TYRA-300). The study involves several groups. In the first group, children aged 5 to 10 will start the medicine right away to help find safe dose levels. Other groups, including children aged 3 to 10, will first have a 6-month observation period where researchers will monitor their usual growth without treatment. After this, they will begin taking the medicine.
During the study, your child will have regular visits to the clinic for check-ups, assessments, and measurements. These will help doctors understand how their bones are growing and if the medicine is causing any side effects. All participants will have the option to continue taking dabogratinib for up to 36 months as part of this study. After that, there might be a chance to continue the treatment in a separate long-term study to see its effects over an even longer period. The study is expected to run from June 2025 until March 2031, so your child would be involved for a significant period.
Risques et avantages potentiels
Emplacements (7)
- Hôpital Great Ormond StreetApproximatifLondon, England
- Sheffield Childrens HospitalVille uniquementSheffield, England
- Guys and St Thomas HospitalVille uniquementLondon, England
- Children's Hospital ColoradoApproximatifAurora, Colorado, United States of America
- Nemours Alfred I duPont Hospital for ChildrenApproximatifWilmington, Delaware, United States of America
- Washington UniversityApproximatifSt Louis, Missouri, United States of America
- Royal Children's HospitalApproximatifParkville, Victoria, Australia
Questions fréquentes
What is achondroplasia?
Achondroplasia is a genetic condition that affects bone growth, leading to shorter limbs and typically shorter height.
What are 'growth plates'?
Growth plates are soft areas at the ends of children's long bones that allow them to grow taller. They close when a child stops growing.
What does a 'Phase II study' mean?
A Phase II study is an early stage of research where a new medicine is tested in people to see if it's safe and if it shows promise for treating a condition, and to find the right dose.
Will my child definitely get the new medicine?
Yes, in this particular study, all participants will receive the new medicine dabogratinib, although some groups will have an observation period first.
How long will my child be in the study?
Your child could be in the study taking the medicine for up to 36 months, with the possibility of continuing in a further long-term study after that.
Comment en savoir plus
Arminder Gandhum
Parlez toujours à votre médecin généraliste ou spécialiste avant de décider de participer à une étude.
Intéressé à participer ?
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