A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
This study is testing a new medicine called vosoritide for babies and toddlers up to three years old who have a condition called hypochondroplasia. Hypochondroplasia affects how bones grow, making someone shorter. We want to find out if vosoritide is safe for these young children and if it can help them grow more. Some children will get the new medicine, and others will get a dummy medicine (placebo), so we can compare the results fairly. The treatment involves a daily injection for about a year, followed by a check-up to make sure everything is okay.
Em resumo
Sobre o que é este estudo?
This study is focusing on a condition called hypochondroplasia, which is a type of dwarfism. It causes bones to develop differently, leading to shorter arms, legs, and a shorter overall height. We’re particularly interested in very young children, from birth up to their third birthday, who have this condition. The main goal is to see if a new medicine, vosoritide, can safely help these children grow taller.
To do this, we'll compare vosoritide with a 'placebo', which is a dummy medicine that looks just like the real one but has no active ingredients. This helps us understand if any changes we see are truly due to the vosoritide or if they might happen anyway. Half the children in the study will get vosoritide, and the other half will get the placebo. Neither the families nor the doctors will know who is getting which, to keep the study fair and unbiased.
This is a 'Phase 2' study, meaning it's an early stage in testing a new medicine. We're trying to understand more about its safety and how well it works before it could potentially be used more widely. The study will last about a year, with daily treatments and follow-up checks to ensure the children are safe and to measure any changes in their growth.
Pontos principais
- Study for children aged 0 to under 3 years with hypochondroplasia.
- Compares a new medicine, vosoritide, with a dummy medicine (placebo).
- Focuses on safety and whether vosoritide improves growth.
- Involves daily injections for about one year.
- A participação é voluntária, e você pode se retirar a qualquer momento.
Quem pode ser elegível?
This study is specifically for babies and toddlers with hypochondroplasia who are aged from birth up to their third birthday. To join, your child must have had a genetic test that confirms they have hypochondroplasia.
Their height will also be checked to make sure they are shorter than what's typical for their age and gender, based on standard growth charts used by doctors. We also need to make sure your child weighs at least 3 kilograms (about 6.6 pounds) before starting.
There are also some reasons why a child might not be able to join. For example, if they have certain other health conditions, have recently taken specific medications like growth hormones, or have an unstable medical issue that might need an operation soon. If your child has ever had cancer, or is currently being checked for it, they wouldn't be able to participate either. Also, if there's any concern about allergic reactions to the study medicine, or if the doctors feel the child won't be able to complete the study safely, they might not be eligible.
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.
- Is my child between birth and their third birthday?
- Does my child have a confirmed genetic diagnosis of hypochondroplasia?
- Is my child significantly shorter for their age and gender?
- Does my child weigh at least 3 kilograms?
- Does my child have any other serious medical conditions or take specific medications?
O que envolve a participação?
If your child takes part, they will receive either the study medicine, vosoritide, or a dummy medicine (a placebo). This will be given once a day as an injection under the skin, similar to how insulin is given. This daily treatment will last for 52 weeks, which is about one year.
Throughout the study, your child will have regular visits to the clinic for health checks, measurements (like height and weight), and to ensure they are handling the treatment well. After the year of daily injections, there will be one final check-up visit about two weeks later to make sure everything is okay. The entire study participation, including the treatment and follow-up, will last approximately 54 weeks.
Riscos e benefícios potenciais
Locais (26)
- Phoenix Children's Hospital - Thomas Campus (Main)Código postal verificadoPhoenix, Estados Unidos· Recrutamento
- Cedars-Sinai Medical CenterCódigo postal verificadoLos Angeles, Estados Unidos· Ainda não recrutando
- Benioff Children's Hospital - OaklandCódigo postal verificadoOakland, Estados Unidos· Recrutamento
- Children's National Medical CenterCódigo postal verificadoWashington D.C., Estados Unidos· Recrutamento
- Ann & Robert H. Lurie Children's Hospital of ChicagoCódigo postal verificadoChicago, Estados Unidos· Recrutamento
- The Johns Hopkins University School of MedicineCódigo postal verificadoBaltimore, Estados Unidos· Recrutamento
- University of Minneasota Masonic Children's HospitalCódigo postal verificadoMinneapolis, Estados Unidos· Recrutamento
- University of MissouriCódigo postal verificadoColumbia, Estados Unidos· Recrutamento
- Cook Children's EndocrinologyCódigo postal verificadoFort Worth, Estados Unidos· Ainda não recrutando
- Children's Wisconsin - Fox Valley HospitalCódigo postal verificadoNeenah, United States· Ainda não recrutando
- Children's Health Queensland Hospital and Health ServiceCódigo postal verificadoSouth Brisbane, Australia· Ainda não recrutando
- Royal Children's Hospital MelbourneCódigo postal verificadoParkville, Austrália· Recrutamento
Perguntas frequentes
What is hypochondroplasia?
Hypochondroplasia is a condition that affects how bones grow, leading to shorter stature (dwarfism), especially in the arms and legs.
What does a 'placebo' mean in this study?
A placebo is a dummy medicine that looks exactly like the real medicine but contains no active ingredients. It helps us compare the effects of the new medicine fairly.
Will my child know if they are getting the real medicine or the placebo?
Neither you, your child, nor the study doctors will know who is getting the real medicine or the placebo until the study is over. This is to ensure the results are fair and unbiased.
Como o medicamento é administrado?
The medicine is given as a daily injection just under the skin, similar to how insulin is administered.
Quanto tempo durará o estudo?
The treatment part of the study will last about one year (52 weeks), followed by a final check-up after two weeks, making the total participation around 54 weeks.
Como saber mais
Trial Specialist
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