A proof of concept study with NVD-003, an autologous osteogenic bone graft, in the treatment of congenital pseudarthrosis of the tibia in pediatric patients.
This study is looking at a new treatment, NVD-003, for children who have a rare condition called congenital pseudarthrosis of the tibia. This is where a bone in the lower leg (the tibia) doesn't heal properly after a break. The treatment uses the child's own bone cells to help the tibia grow and strengthen. The study is in its early stages (Phase I and II), focusing on how safe the treatment is and if it helps the bone mend. Researchers will check for any side effects and how well the bone heals over 24 months, using scans and observing the child's progress.
At a glance
What is this study about?
This study is about a new way to treat a bone problem called congenital pseudarthrosis of the tibia, which affects children. In this condition, the shin bone (tibia) doesn't heal properly and can be very weak or even break. The specific treatment being tested is called NVD-003. This is quite special because it uses the child's own bone-building cells to create a new bone graft, which is then put into the leg to help the weak bone heal and become stronger.
The main goal of this study is to find out if NVD-003 is safe for children. The researchers will closely watch for any bad reactions or side effects that might happen after the treatment. They will also look at how well the new bone graft works to help the tibia mend. They want to see if it encourages the bone to grow and if it becomes strong enough for the child to use their leg normally.
Over two years, doctors will use different methods to check on the child's progress. This includes regular scans like CT scans and X-rays to see how the bone is healing and growing. They will also assess the child's daily activities, like walking and playing, to understand how the treatment affects their overall well-being. This kind of research is really important to develop better treatments for children with this challenging condition.
Key takeaways
- Tests a new treatment for congenital pseudarthrosis of the tibia.
- Uses a child's own bone cells (NVD-003) to help bone heal.
- Focuses on checking safety and bone healing over 2 years.
- Involves regular hospital visits, scans, and physical assessments.
- Aims to find better ways to treat this rare bone condition.
Who may be eligible?
This study is open to both boys and girls of all ages who have congenital pseudarthrosis of the tibia. There are no specific age limits mentioned, meaning that children of various ages with this bone condition could potentially be considered.
To join, a child must have received a diagnosis of congenital pseudarthrosis of the tibia from their doctor. This study is specifically looking for children with this particular condition. Doctors will carefully review each child's medical history to make sure the study treatment is suitable for them.
Because this uses a child's own cells, there will likely be other medical checks to ensure they are healthy enough for the procedure. The medical team will discuss all the requirements with you, ensuring you understand if your child can take part.
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.
- Does my child have congenital pseudarthrosis of the tibia?
- Is my child of any age? (There are no specific age limits mentioned.)
- Is my child generally well enough to undergo surgery?
- Am I able to commit to regular hospital visits over two years?
What does participation involve?
If your child takes part, the study will involve several visits to the hospital over a period of 24 months (two years). It starts with an initial check-up to see if your child is suitable. If they are, they would have the NVD-003 treatment, which involves a surgery to place the bone graft.
After the surgery, there will be follow-up appointments at various times: at hospital discharge, then at 6 weeks, and at 3, 6, 12, and 24 months after the surgery. These visits will include different assessments. Doctors will take X-rays and CT scans to see how the bone is healing and growing. They will also check on your child's general well-being, movement, pain levels, and how they manage activities like walking and playing, using special assessment tools. The total duration of the study for your child would be around 24 months.
Potential risks and benefits
Locations (1)
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Common questions
What is congenital pseudarthrosis of the tibia?
It's a rare condition where the main bone in the lower leg (the tibia) is very weak and doesn't heal properly, often breaking easily.
What is NVD-003?
NVD-003 is an experimental treatment that uses a child's own bone cells to create a special bone graft to help the tibia heal.
How long will my child be in the study?
Your child would be part of the study for about two years (24 months), with several hospital visits and check-ups during that time.
What kind of tests will my child have?
They will have X-rays and CT scans to check bone healing, and doctors will also assess their movement, pain, and how they do daily activities.
Can I take my child out of the study?
Yes, you can choose to withdraw your child from the study at any time, and this will not affect their regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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