A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
This clinical trial is testing a new medicine called navepegritide for young people aged 12 to 18 who have achondroplasia. Achondroplasia is a condition that affects bone growth, leading to shorter stature. In this study, we want to find out if navepegritide can help teenagers grow more and if it's safe to use. Half the participants will receive weekly injections of the active medicine, while the other half will receive a 'dummy' medicine (placebo) that looks the same but contains no active drug. We will measure how much participants grow over 52 weeks. This is a Phase 2 study, meaning it's still in the earlier stages of testing.
At a glance
What is this study about?
This study is investigating a new medicine called navepegritide for teenagers between 12 and 18 years old who have achondroplasia. Achondroplasia is the most common type of dwarfism, caused by a change in a specific gene that affects how bones grow. This can lead to shorter arms and legs, and a shorter stature overall. The main goal of this research is to see if navepegritide can help these young people grow taller and if it is safe for them to use.
To find out if the medicine works, we will be comparing navepegritide with a 'placebo'. A placebo is a dummy medicine that looks exactly like the real medicine but has no active ingredients. This helps scientists understand if any changes seen are truly due to the medicine being tested, or if they might happen anyway. Participants will receive weekly injections, and we will carefully measure their growth over a period of 52 weeks.
This is a 'Phase 2' trial, which means it's one of the earlier steps in testing a new medicine. Before any medicine can be used more widely, it has to go through several phases of trials to make sure it's safe and effective. If this study shows promising results, it could potentially lead to further research and, eventually, a new treatment option for young people with achondroplasia.
Key takeaways
- This study is testing a new medicine, navepegritide, for achondroplasia.
- It involves teenagers aged 12-18 and compares the medicine to a dummy pill (placebo).
- The main goal is to see if the medicine helps with growth and if it's safe.
- Participation includes weekly injections and regular clinic visits over 52 weeks.
- It's a Phase 2 trial, meaning it's still an early stage of testing.
Who may be eligible?
To join this study, participants must be between 12 and 18 years old. They need to have a confirmed diagnosis of achondroplasia, which has been shown through a genetic test. Also, a parent or legal guardian must be willing and able to give weekly injections of the study medicine and follow all the study instructions carefully.
There are also some reasons why someone might not be able to join. For example, if they have recently taken part in another clinical trial where they received a study medicine, or if their growth has already slowed down a lot (less than 1.5 cm per year) or their growth plates have closed. We also can't include anyone who has another medical condition that causes short stature, or who is allergic to any of the ingredients in the study medicine.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you between 12 and 18 years old?
- Do you have a genetically confirmed diagnosis of achondroplasia?
- Is a parent/guardian willing to help with weekly injections?
- Have you grown at least 1.5 cm in the last year, and are your growth plates not closed?
- Do you have any other conditions that cause short stature?
What does participation involve?
If you decide to take part, you would receive weekly injections of either the study medicine (navepegritide) or a dummy medicine called a placebo. These injections would be given just under the skin by a parent or guardian. The study will last for 52 weeks, which is about one year.
During this year, you would need to visit the study clinic regularly. At these visits, doctors and nurses will take measurements, ask about your health, and perform check-ups to see how you are doing and track your growth. There will be blood tests and other assessments to monitor your health and safety. The study team will explain exactly what each visit involves and how often they will be.
Potential risks and benefits
Locations (5)
- Ascendis Investigational SiteVerified postcodeMontreal, Canada· Recruiting
- Ascendis Investigational SiteVerified postcodeCopenhagen, Denmark· Recruiting
- Ascendis Investigational SiteVerified postcodeParis, France· Recruiting
- Ascendis Investigational SiteVerified postcodeDublin, Ireland· Recruiting
- Ascendis Investigational SiteVerified postcodeNorwich, United Kingdom· Recruiting
Common questions
What is achondroplasia?
Achondroplasia is a condition that affects bone growth, leading to shorter stature, especially in the arms and legs. It's caused by a genetic change.
What is navepegritide?
Navepegritide is a new medicine being tested to see if it can help improve growth in teenagers with achondroplasia.
What is a 'placebo'?
A placebo is a dummy medicine that looks exactly like the real medicine but doesn't contain any active drug. It helps researchers understand if the real medicine is actually working.
How long does the study last?
The main treatment part of this study lasts for 52 weeks, which is about one year.
What does 'Phase 2' mean?
'Phase 2' means this is an earlier stage of testing a new medicine. We're looking to see if it's safe and if it shows signs of working.
How to find out more
Ascendis Registry Inquiries
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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