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.
Överblick
Vad handlar denna studie om?
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.
Viktiga punkter
- 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.
- Deltagande är frivilligt, och du kan dra dig ur när som helst.
Vem kan vara berättigad?
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.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- 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?
Vad innebär deltagande?
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.
Potentiella risker och fördelar
Platser (26)
- Phoenix Children's Hospital - Thomas Campus (Main)Verifierat postnummerPhoenix, USA· Rekrytering
- Cedars-Sinai medicinska centrumVerifierat postnummerLos Angeles, USA· Rekryterar inte ännu
- Benioff Children's Hospital - OaklandVerifierat postnummerOakland, USA· Rekrytering
- Nationellt medicinskt center för barnVerifierat postnummerWashington D.C., USA· Rekrytering
- Ann & Robert H. Lurie Barnsjukhus i ChicagoVerifierat postnummerChicago, USA· Rekrytering
- The Johns Hopkins University School of MedicineVerifierat postnummerBaltimore, USA· Rekrytering
- University of Minneasota Masonic Children's HospitalVerifierat postnummerMinneapolis, USA· Rekrytering
- University of MissouriVerifierat postnummerColumbia, United States· Rekrytering
- Cook Children's EndocrinologyVerifierat postnummerFort Worth, United States· Rekryterar inte ännu
- Children's Wisconsin - Fox Valley HospitalVerifierat postnummerNeenah, United States· Rekryterar inte ännu
- Children's Health Queensland Hospital and Health ServiceVerifierat postnummerSödra Brisbane, Australien· Rekryterar inte ännu
- Royal Children's Hospital MelbourneVerifierat postnummerParkville, Australien· Rekrytering
Vanliga frågor
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.
Hur ges medicinen?
The medicine is given as a daily injection just under the skin, similar to how insulin is administered.
Hur länge kommer studien att pågå?
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.
Hur du får reda på mer
Trial Specialist
Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.
Intresserad av att delta?
Diskussion
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