A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia
This clinical trial is exploring a new treatment approach for children with achondroplasia, a condition that affects bone growth. The study focuses on children aged 2 to 11 years and combines two medicines: navepegritide and lonapegsomatropin. Both are given as separate injections under the skin once a week. The main goal is to find out if using these two medicines together is effective, safe, and well-tolerated. This is a "proof-of-concept" study, meaning it's an early step to see if this combination treatment shows promise. It's hoped that this combination could help children with achondroplasia grow better and improve their overall health.
At a glance
What is this study about?
This study is an important step in exploring new ways to help children with achondroplasia. Achondroplasia is a lifelong condition that affects how bones grow, leading to short stature and other health challenges. Doctors and researchers are always looking for better treatments to improve the health and well-being of children with this condition.
This particular study is trying something new: combining two medicines that are already known, called navepegritide and lonapegsomatropin. Researchers want to see if giving these two medicines together works better than giving just one. This is a "Phase 2" trial, which means it's an important early stage of research. It helps scientists understand if the new treatment approach is effective, safe, and tolerated by the body before moving on to larger studies.
The idea is to give both medicines as separate injections under the skin once a week to children aged 2 to 11. By combining them, the hope is to boost the positive effects on growth and health. This type of research helps us learn more about achondroplasia and potentially find new ways to support children living with the condition.
Key takeaways
- This study is for children with achondroplasia aged 2-11.
- It tests a new combination of two weekly injectable medicines.
- The goal is to see if this combination is safe and helps with growth.
- Parents will be trained to give injections at home.
- Regular clinic visits are part of the study to monitor health and progress.
- Participation allows children to access a potential new treatment approach.
Who may be eligible?
To join this study, we're looking for children with achondroplasia between the ages of 2 and 11. It's important that their achondroplasia has been confirmed by a genetic test, and they must be able to stand on their own without help. Their parents or guardians must be willing and able to give weekly injections and follow all the study's instructions carefully.
There are also some reasons why a child might not be able to join. For example, if they've taken part in another clinical trial recently (unless it was for specific achondroplasia studies), or if their growth plates (the areas where bones grow) have already closed. Children with certain allergies to the medicines being studied, or other medical conditions that affect growth besides achondroplasia, wouldn't be able to participate. Also, if they're currently taking other medicines that influence growth, they might not be suitable.
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.
- Is your child between 2 and 11 years old?
- Does your child have a confirmed diagnosis of achondroplasia?
- Can your child stand on their own without help?
- Are you able and willing to give weekly injections and attend clinic visits?
- Has your child avoided other clinical trials (other than specific achondroplasia studies) recently?
- Has your child not received other medicines intended to affect growth?
What does participation involve?
If your child joins this study, they will receive two different medicines, navepegritide and lonapegsomatropin. These will be given as separate injections under the skin, once a week. Your child's parent or guardian will be trained on how to give these injections at home. There will be regular visits to the clinic so the study team can check on your child's health, measure their growth, and make sure the treatment is going well. These visits will also involve checking on their general health through blood tests and other assessments. The study will last for a period of time, including a follow-up period after the treatment ends, to monitor your child's progress and safety.
Potential risks and benefits
Locations (3)
- Ascendis Pharma Investigational SiteVerified postcodeCopenhagen, Denmark
- Ascendis Pharma Investigational SiteVerified postcodeDublin, Ireland
- Ascendis Pharma Investigational SiteVerified postcodeLondon, United Kingdom
Common questions
What is achondroplasia?
Achondroplasia is a genetic condition that affects bone growth, leading to short stature and other specific features.
What are Navepegritide and Lonapegsomatropin?
These are two medicines that, in this study, are being tested together to see if they can help improve growth in children with achondroplasia.
How are the medicines given?
The medicines are given as injections under the skin, once a week, by a parent or guardian at home.
What is a 'Phase 2' trial?
A Phase 2 trial is an early stage of research that helps doctors find out if a new treatment approach is effective, safe, and well-tolerated.
Can my child stop participating if we change our minds?
Yes, you and your child can choose to stop participating in the study at any time, for any reason.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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