RecruitingPHASE3INTERVENTIONAL

A Study to Assess How Effective and Safe NVD003 is for Treating Patients With Congenital Pseudarthrosis of the Tibia.

This research study is about finding a better way to treat children who have a difficult-to-heal broken bone in their lower leg. This condition is called Congenital Pseudarthrosis of the Tibia, or CPT. In CPT, the shin bone (tibia) doesn't heal properly, often after a minor injury, and can lead to many surgeries. The study is comparing a new treatment called NVD003 with a common surgery where bone is taken from the hip (iliac crest bone graft). NVD003 is a special material created to help new bone grow. Doctors want to see if NVD003 is more effective and safer than the current standard treatment for children with CPT, helping their bones connect and stay strong.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Novadip Biosciences

Enrolment target

Start

02 Sep 2025

Estimated completion

01 Jul 2027

Congenital Pseudarthrosis of Tibia

What is this study about?

Congenital Pseudarthrosis of the Tibia (CPT) is a rare condition that affects children, usually discovered in their first year of life. It means that the shin bone (tibia) in one leg doesn't heal after it breaks, or sometimes it breaks easily in an area that wasn't strong to begin with. This can be a very challenging problem to treat, as traditional surgeries often don't work the first time, leading to many operations and, in some cases, even the need to remove part of the leg.

Currently, one common treatment involves taking a piece of bone from the child's hip (called an iliac crest bone graft) and using it to try and fix the broken shin bone. However, this method doesn't always succeed in helping the bone heal permanently, and it can also involve a second surgery site. This study is looking at a new treatment called NVD003. NVD003 is a specially designed material made from the child's own fat cells combined with other bone-like particles. The idea behind NVD003 is that it offers a different way to help the bone grow and heal, even in these difficult CPT cases.

The main goal of this study is to see if NVD003 is more effective than the standard hip bone graft in helping children with CPT achieve a strong, healed shin bone. Doctors also want to make sure NVD003 is safe. By comparing the two treatments, researchers hope to find a better, more reliable way to help children with CPT avoid multiple surgeries and improve their long-term health.

Key takeaways

The study is for children aged 17 or under with CPT.
It compares a new treatment (NVD003) with standard hip bone graft surgery.
The goal is to find out if NVD003 is more effective and safer for healing CPT.
Participation involves surgery and follow-up visits over 12 months.
You can take your child out of the study at any time if you wish.

Who may be eligible?

This study is looking for children who are 17 years old or younger who have been diagnosed with Congenital Pseudarthrosis of the Tibia (CPT). They must have a specific type of shin bone fracture (called Paley type 3 or 4) that isn't healing on its own and are planning to have surgery with an internal rod.

Your child can participate if a parent or legal guardian gives written permission for them to join. Depending on your child's age, they might also need to give their own OK to join. Before starting, doctors will need to do some blood tests to make sure your child doesn't have certain viruses (like HIV or Hepatitis B/C) or other infections.

Also, your child must weigh at least 5 kg (about 11 pounds). If your child is female and old enough to have periods, she must not be pregnant or breastfeeding, and must be using very effective birth control methods during the study period. This is to ensure her safety and the accuracy of the study results.

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 17 years old or younger?
Does your child have a diagnosis of Congenital Pseudarthrosis of the Tibia (CPT)?
Does your child need surgery for a difficult-to-heal shin bone fracture?
Does your child weigh at least 5 kg (about 11 pounds)?
If female, is your child not pregnant or breastfeeding, and using effective birth control if of childbearing age?

Answer every question to see your result.

What does participation involve?

If your child takes part, they will either receive the new treatment (NVD003) or the standard bone graft surgery from their hip. Both treatments involve surgery where an internal rod is used to help fix the bone. The doctors will compare how well the bones heal and check for any side effects over 12 months after the surgery.

Throughout the study, there will be several visits to the clinic for check-ups. These visits will include exams, X-rays to check bone healing, and blood tests. You and your child will be asked about your child's general health and any changes. We don't have exact details on the frequency of visits or specific tests, but the total active study period for comparing the main treatments is 12 months after surgery.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of this study is that your child might receive a new treatment (NVD003) that could be more effective at healing their CPT and potentially lead to fewer surgeries compared to the current standard treatment. However, as it's a new treatment, not all risks are fully known, and there might be side effects from either NVD003 or the standard surgery, including risks associated with anaesthesia and surgery itself, infection, or the bone not healing as expected. You have the right to withdraw your child from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (7)

Phoenix Children's Hospital, Inc.
Verified postcode
Phoenix, United States· Recruiting
Loma Linda University Health
Verified postcode
Loma Linda, United States· Recruiting
LifeBridge Health - International Center for Limb Lengthening
Verified postcode
Baltimore, United States· Recruiting
Mayo Clinic
Verified postcode
Rochester, United States· Recruiting
Cliniques Universitaires Saint-Luc ASBL (CUSCL)
Verified postcode
Brussels, Belgium· Recruiting
CHU Amiens-Picardie
Verified postcode
Amiens, France· Recruiting
Hospital Sant Joan de Déu
Verified postcode
Esplugues de Llobregat, Spain· Recruiting

Common questions

What is CPT?

CPT stands for Congenital Pseudarthrosis of the Tibia. It's a rare condition where the shin bone (tibia) in a child's lower leg doesn't heal correctly after a break, or might break very easily.

What is NVD003?

NVD003 is a new treatment being tested. It's a special material made from a child's own fat cells and other tiny particles, designed to help new bone grow and heal the shin bone.

Will my child definitely get the new treatment?

No, participants will be randomly assigned to either receive NVD003 or the standard bone graft surgery from the hip. This is so researchers can fairly compare the two treatments.

How long will the study last for my child?

The main part of the study assessing the treatments will last for 12 months following your child's surgery.

What if we change our minds about participating?

You are free to withdraw your child from the study at any time, for any reason, and it will not affect their regular medical care.

How to find out more

Judy Ashworth, MD

clinical@novadip.com 224-713-6154 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.