A Study of TYRA-300 in Children With Achondroplasia: BEACH301
The BEACH301 study is exploring a new medication called TYRA-300 for children aged 3 to 10 who have achondroplasia. Achondroplasia is a condition that affects how bones grow, leading to shorter limbs. This study aims to understand if TYRA-300 is safe for children, how well their bodies handle it, and to identify the most effective dose. The children taking part still have 'open growth plates', which are areas in their bones where growth happens. This is a Phase 2 study, meaning it's an early step to gather more information after initial safety tests. The goal is to see if this medicine could help children with achondroplasia grow more effectively.
De un vistazo
¿De qué trata este estudio?
This study, known as BEACH301, is testing a new medicine called TYRA-300 for children who have a condition called achondroplasia. Achondroplasia is a genetic condition that affects how bones grow, particularly in the arms and legs, leading to shorter limbs. The study includes children aged 3 to 10 years old who still have 'open growth plates' – these are parts of their bones that are still growing.
The main goals of this study are to see if TYRA-300 is safe for children to take, how well their bodies tolerate it (meaning if they have uncomfortable side effects), and to figure out the right amount of medicine (the dose) that might be helpful. This is a 'Phase 2' study, which means it’s an important step in researching new medicines. It helps us learn more about the medicine's effects in people after initial safety checks.
Researchers are looking at different doses of TYRA-300 in this study. They hope to find a dose that could potentially help children with achondroplasia grow. The study is designed to carefully observe how children respond to the medicine and to gather crucial information for its future development.
Puntos clave
- This study is for children aged 3-10 with achondroplasia.
- It tests a new medicine, TYRA-300, taken by mouth.
- The main goals are to check safety, tolerability, and find the right dose.
- Children must have 'open growth plates' to join.
- Parents and children must agree to participate.
- Participation involves regular clinic visits and monitoring.
¿Quién puede ser elegible?
To join this study, children need to be between 3 and 10 years old. They must have a confirmed diagnosis of achondroplasia and still have 'open growth plates,' which are the areas in their bones that allow them to grow. It's also important that they can stand and walk by themselves and can take medicine by mouth.
There are a few specific groups within the study. Some children (aged 5-10) will be in a 'safety group' first. Others (aged 3-10) will either not have had any previous growth-boosting treatments, or they will have had such treatments in the past. Parents or legal guardians must give their permission for their child to take part, and if old enough, the child will also be asked if they want to participate.
Children cannot join the study if they have other serious health conditions that might interfere with the study or their growth, or if they have certain eye problems. Those who have had or plan to have limb-lengthening surgery, or who are taking certain other medications, also won't be able to participate.
¿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 between 3 and 10 years old?
- Does your child have a confirmed diagnosis of achondroplasia?
- Does your child's doctor confirm they have 'open growth plates'?
- Can your child stand and walk on their own?
- Can your child take medicine by mouth?
- Has your child not had limb-lengthening surgery or planned for one soon?
¿En qué consiste la participación?
If your child takes part in this study, they will receive the study medicine, TYRA-300, which is taken by mouth. The study involves several visits to a clinic where doctors and nurses will check your child's health, measure their growth, and take blood samples to see how the medicine is working and to monitor for any side effects. These visits will help the researchers understand how safe the medicine is and what dose might be most effective.
Your child will be carefully monitored throughout the study. The study duration is not specified in detail, but it will involve regular check-ups and follow-up appointments. The medical team will explain the full schedule of visits and assessments before you decide to participate.
Riesgos y beneficios potenciales
Locations (25)
- Lundquist Institute for Biomedical InnovationCódigo postal verificadoTorrance, United States· Reclutamiento
- Hospital de Niños de ColoradoCódigo postal verificadoAurora, Estados Unidos· Reclutamiento
- Nemours Alfred I duPont Hospital for ChildrenCódigo postal verificadoWilmington, United States· Reclutamiento
- Johns Hopkins University School of MedicineCódigo postal verificadoBaltimore, Estados Unidos· Reclutamiento
- Uncommon CuresCódigo postal verificadoChevy Chase, United States· Reclutamiento
- University of MissouriCódigo postal verificadoColumbia, United States· Reclutamiento
- Washington UniversityCódigo postal verificadoSan Luis, Estados Unidos· Reclutamiento
- Duke University HospitalCódigo postal verificadoDurham, Estados Unidos· Reclutamiento
- Vanderbilt University Medical CenterCódigo postal verificadoNashville, Estados Unidos· Reclutamiento
- Children's Medical Center, DallasCódigo postal verificadoDallas, United States· Reclutamiento
- University of Texas Health Science Center Medical School at HoustonCódigo postal verificadoHouston, Estados Unidos· Reclutamiento
- University of Wisconsin-MadisonCódigo postal verificadoMadison, United States· Reclutamiento
Preguntas comunes
What is achondroplasia?
Achondroplasia is a genetic condition that affects bone growth, leading to shorter arms and legs.
What does 'open growth plates' mean?
It means the areas in your child's bones where growth normally happens are still active and open.
Is TYRA-300 a pill?
Yes, TYRA-300 is a medicine that children will take by mouth.
What is a 'Phase 2' study?
It's an early stage of testing a new medicine in people to see if it's safe and if it works.
Will my child definitely grow taller in this study?
There's no guarantee the medicine will work for every child, as this study is still trying to find out if it is effective.
Cómo obtener más información
Lara Balick
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
¿Interesado en participar?
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Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.