A Phase 2, open-label, multi-center, 2-stage sequential cohort, dose escalation study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of subcutaneous SAR442501 in pediatric participants with Achondroplasia
This research is a Phase 2 study looking at a new medicine called SAR442501 for children and young people with achondroplasia, which is a condition affecting bone growth, leading to short stature. The medicine is given by injection under the skin. Researchers want to find out if this medicine is safe, how well people tolerate it, and how the body processes it. They are also exploring if it helps improve growth, affects bone development, and improves overall quality of life. This is an open-label study, meaning both participants and researchers will know who is receiving the medicine. It's a key step in understanding if this treatment could be helpful for those living with achondroplasia.
Överblick
Vad handlar denna studie om?
This study is focused on a genetic condition called achondroplasia, which is the most common form of dwarfism. It affects how bones grow, particularly in the arms and legs, leading to a shorter stature and sometimes other health challenges. Researchers are testing a new medicine, SAR442501, to see if it can help improve the growth of bones in children and young people with this condition. This medicine is given as an injection under the skin, similar to how some people might take insulin.
The main goal of this study is to check the safety of the new medicine. This means carefully watching for any side effects or unwanted reactions. The researchers will also look at how the body handles the medicine – how it's absorbed, distributed, used, and removed. Beyond safety, they're hoping to understand if the medicine has any positive effects on growth, such as changes in height or the proportions of different body parts. They will also look at whether it improves overall well-being and reduces pain.
This is a 'Phase 2' study, which means it's one of the earlier stages of testing a new medicine. It's designed to gather more detailed information about the medicine's effects and find the right dose before potentially moving on to larger studies. The aim is to develop a treatment that could make a real difference to the lives of people with achondroplasia.
Viktiga punkter
- This study is testing a new medicine for achondroplasia.
- The medicine is given as an injection under the skin.
- The main goals are to check safety and see if it helps with growth.
- It's an early-stage study, and more research will be needed.
- Du har rätt att lämna studien när som helst.
Vem kan vara berättigad?
This study is looking for children and young people who have been diagnosed with achondroplasia. Importantly, the study is open to both boys and girls of all ages.
While the information provided doesn't list specific reasons why someone *wouldn't* be able to join, generally, studies have rules about other health conditions, medicines you might be taking, or if you've recently been part of another trial. These rules are in place to ensure the safety of everyone taking part and to make sure the study results are clear and accurate.
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.
- Do you or your child have a confirmed diagnosis of achondroplasia?
- Is the person considering joining a child or young person (all ages are welcome)?
- Kan du delta i regelbundna klinikbesök?
- Are you comfortable with the idea of injections and blood tests?
Vad innebär deltagande?
If you decide to take part in this study, the main part of it will involve receiving regular injections of the study medicine, SAR442501, under the skin. Your child will have these injections over a period of time, and both you and the study team will know that they are receiving the active medicine.
You can expect regular visits to the study clinic during this time. At these visits, the study team will carefully monitor your child's health and look for any side effects. They will also take measurements, such as height, arm and leg lengths, and head circumference. Blood tests will be taken to see how the medicine is working in the body and to check for any changes in bone markers. You may also be asked to fill out questionnaires about your child's quality of life, pain levels, and their development. The total duration of the study is not specified but will involve multiple assessments over time.
Potentiella risker och fördelar
Platser (3)
- —ObekräftadItalien
- —ObekräftadSpanien
- —ObekräftadTjeckien
Vanliga frågor
What is achondroplasia?
Achondroplasia is a genetic condition that affects how bones grow, leading to shorter arms and legs, and shorter stature.
What does 'Phase 2 study' mean?
Phase 2 means it's an early-stage study, checking the safety of the new medicine and seeing if it has any positive effects, usually in a small group of people.
Hur ges medicinen?
The medicine, SAR442501, is given as an injection just under the skin.
Will we know if my child is getting the actual medicine?
Yes, this is an 'open-label' study, meaning everyone involved will know that the study medicine is being given.
Vilka typer av tester kommer att utföras?
Tests will include checking for side effects, measuring growth, taking blood samples, and filling out questionnaires about health and well-being.
Hur du får reda på mer
Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.
Intresserad av att delta?
Diskussion
Gemenskapsdiskussion
Drivs av vårt forum på community.patient.info. Var vänlig och respektfull — detta är inte medicinsk rådgivning.