Ongoing Clinical Trials for Congenital Pseudarthrosis
There are currently 2 ongoing clinical trials investigating new treatment options for congenital pseudarthrosis of the tibia in children. These trials are testing an innovative stem cell-based bone graft called NVD003, comparing it to traditional bone grafting methods. The studies are taking place in Belgium, France, and Spain.
Clinical trial locations
- Belgium
- France
- Spain
Study of NVD003 stem cell implant compared to bone graft for children with congenital pseudarthrosis of the tibia
This trial focuses on children with congenital pseudarthrosis of the tibia, a rare condition where the shin bone develops an abnormal connection that doesn’t heal properly. The condition typically becomes apparent when a child begins to bear weight on the affected leg, causing the weakened bone to break. Without intervention, the bone fragments fail to unite properly, leading to instability in the lower leg.
Main inclusion criteria: Children aged 17 years or younger who weigh at least 5 kg can participate in this study. They must have a confirmed diagnosis of congenital pseudarthrosis of the tibia with a non-healing fracture classified as type 3 or 4 in the middle portion of the bone. The child must be suitable for surgery using an internal rod to stabilize the bone. Parents or legal guardians must provide written consent, and the child must give agreement if age-appropriate. All participants must test negative for infectious diseases including HIV and hepatitis. If the child is old enough to have children, effective birth control methods must be used throughout the study.
Main exclusion criteria: Children cannot participate if they have had any previous surgical procedures to treat this condition. They must not have active infections at the surgical site, blood disorders that could affect bone healing, or any other medical conditions that could interfere with the treatment. Children who are allergic to the study medication or its components, or who are participating in other clinical trials, are excluded. Female participants must not be pregnant or breastfeeding.
Focus and goal: The study will test a treatment called NVD003, which is made from a patient’s own fat-derived stem cells and is surgically implanted into the affected bone area. This treatment will be compared to the current standard procedure that uses bone tissue taken from the hip bone. The purpose is to determine how well NVD003 works and how safe it is when used to treat children with this bone condition. The treatment will be monitored for 12 months to check how well the bone heals. During the study, doctors will take X-rays and perform physical examinations to check bone healing, assess the child’s ability to walk, and evaluate their overall quality of life.
Investigational drug: NVD003 is an investigational treatment being studied as an alternative to the standard procedure of using bone graft taken from the patient’s own hip bone. This treatment aims to help the bone heal properly after surgery by utilizing the patient’s own fat-derived stem cells to promote bone formation and healing.
Study on NVD-003 Bone Graft for Treating Congenital Pseudarthrosis of the Tibia in Children
This clinical trial focuses on the same rare bone condition affecting the shin bone in children. The condition can cause the affected leg to be weaker and more prone to fractures, with the bone not healing correctly over time. This can lead to difficulties in walking and noticeable differences in leg length.
Main inclusion criteria: Children between 2 and 8 years old who weigh at least 5 kg can participate. They must have congenital pseudarthrosis of the tibia with a Paley type 3 or 4 fracture that is not healing. Children can participate whether or not they have the related condition NF1 (neurofibromatosis type 1). Participants can have had no more than 2 previous unsuccessful surgeries to fix the main fracture. The child must be healthy enough to undergo surgery with anesthesia, and it must be possible to collect at least 2cc of fat tissue from them. Test results must show they do not have certain infections. The amount of NVD-003 graft needed should not exceed 20cc. Parents or legal guardians must provide written consent.
Main exclusion criteria: Children with any other serious medical condition that could interfere with the study cannot participate. Those who have had recent surgery, are planning surgery soon, or are currently in another clinical trial are excluded. Children with allergies to study substances, those unable to follow study procedures, or who have infections that could affect results are not eligible. Children with immune system conditions, bleeding disorders that prevent proper blood clotting, or a history of drug or alcohol abuse are also excluded. Pregnant or breastfeeding females cannot participate.
Focus and goal: The study will test NVD-003, an implant made from the patient’s own fat-derived stem cells, which is used to create a bone graft that can help the tibia heal. A small amount of fat tissue will be collected from the patient and used to create the bone graft, which will then be implanted into the affected area of the tibia. The study will monitor patients over approximately two years to ensure the treatment is safe and to observe how well the bone heals. Participants will undergo regular check-ups, including imaging tests like X-rays and CT scans, to track the progress of bone healing. The patient’s general functioning, walking ability, and pain levels will also be assessed at regular intervals.
Investigational drug: NVD-003 is an autologous osteogenic bone graft, meaning it is derived from the patient’s own cells and designed to promote bone growth and healing. At the molecular level, NVD-003 works by utilizing the patient’s own cells to stimulate new bone formation, enhancing the body’s natural healing processes. It is classified as a regenerative medicine product.
Summary
Both ongoing clinical trials are investigating the same innovative treatment approach using NVD-003, a stem cell-based bone graft derived from the patient’s own fat tissue. The trials represent an important advancement in treating congenital pseudarthrosis of the tibia, a challenging condition that often requires multiple surgeries and has limited treatment options.
The first trial is a comparative study being conducted across three countries (Belgium, France, and Spain) and includes children up to 17 years old. It directly compares NVD003 to the traditional bone grafting method using hip bone tissue. The second trial, conducted in Belgium, focuses on a slightly younger age group (2 to 8 years old) and primarily assesses the safety of the NVD-003 treatment over a two-year period.
Both trials use a personalized medicine approach, creating the bone graft from each patient’s own cells, which may reduce the risk of rejection and eliminate the need for taking bone from the hip, a procedure that can cause additional pain and complications. These studies offer hope for improved outcomes in children suffering from this rare and difficult-to-treat bone condition.
