Not recruitingRare disease

Study on NVD-003 Bone Graft for Treating Congenital Pseudarthrosis of the Tibia in Children

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare bone condition called , which affects children. This condition involves a bone in the lower leg, known as the tibia, that does not heal properly after a fracture. The study is testing a new treatment called , which is an implant made from the patient's own fat-derived stem cells. These cells are used to create a bone graft that can help the tibia heal.

The purpose of the study is to assess the safety of the treatment. The study will involve a procedure to collect fat tissue from the patient, which will then be used to create the bone graft. This graft will be implanted into the affected area of the tibia. The study will monitor the patients over a period of time to ensure the treatment is safe and to observe how well the bone heals.

Participants in the study will undergo regular check-ups, including imaging tests like X-rays, to track the progress of the bone healing. The study will last for about two years, during which the safety and effectiveness of the treatment will be closely monitored. This research aims to provide a new option for children with this challenging condition, potentially improving their ability to heal and function normally.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial screening
The initial screening involves a series of tests to confirm eligibility for the trial. This includes checking the patient's medical history and conducting serology and molecular tests to exclude certain infections such as HIV, hepatitis, and syphilis.
The patient's general health condition is assessed to ensure they can safely undergo the required surgical procedures with anesthesia.
Step 2
Adipose tissue collection (ATC)
A small amount of adipose tissue, ranging from 2cc to 10cc, is collected from the patient. This tissue is used to create the NVD-003 graft, which is an autologous osteogenic bone graft.
Step 3
Grafting surgery (GS)
The NVD-003 graft is implanted into the affected area of the tibia. This procedure is performed to treat congenital pseudarthrosis of the tibia, a condition where the bone does not heal properly.
The volume of the graft is determined based on the size of the bone defect, ensuring it does not exceed 20cc.
Step 4
Post-surgery monitoring
Short-term safety is monitored from the time of screening until 3 months after the grafting surgery. This includes observing any adverse effects related to the adipose tissue collection and the NVD-003 graft.
Mid long-term safety is assessed from 3 months to 12 months post-surgery, focusing on the graft implantation and any surgical reinterventions needed.
Long-term safety is evaluated from 12 months to 24 months post-surgery, monitoring any serious adverse effects related to the NVD-003 graft.
Step 5
Follow-up assessments
Regular assessments are conducted to evaluate the clinical progress of the patient. This includes analyzing bone formation and union through CT scans and X-rays at various intervals: 6 weeks, 3 months, 6 months, 12 months, and 24 months post-surgery.
The patient's general functioning, walking ability, and pain levels are assessed using the Clinical Global Impression scales at these same intervals.

Who can join the trial?

13 criteria

The patient must be a boy or girl between 2 and 8 years old.
The patient must weigh at least 5 kg (11 lbs).
The patient must have a condition called **congenital pseudarthrosis of the tibia**, which means a specific type of bone problem in the lower leg.
The patient can have a related condition called **NF1** or not.
The patient must have a type of fracture that is not healing, known as **Paley type 3 or 4 fracture**.
The patient can have had no more than 2 previous unsuccessful surgeries to fix the main fracture.
The patient must be healthy enough to have surgery, including procedures called **ATC** (adipose tissue collection) and **GS** (grafting surgery), according to usual medical care.
The patient must have test results showing they do not have certain infections, including **HTLV1/2**, **HIV 1/2**, **hepatitis B**, **hepatitis C**, and **syphilis**.
It must be possible to collect at least 2cc (0.07 fl. oz.) of fat tissue from the patient, ideally up to 10cc (0.34 fl. oz.).
The patient must be in good general health to safely undergo surgeries with anesthesia.
The patient's fracture and overall condition must allow for a specific surgical procedure as outlined in the study.
The amount of **NVD 003 graft** needed should not be more than 20cc (0.68 fl. oz.), as determined by the surgeon using X-ray measurements. This amount should be enough to fill the bone gap in the tibia and any related bone areas.
The patient's parent(s) or legal guardian(s) must provide written consent for the patient to join the study.

Who cannot join the trial?

10 criteria

Patients with any other serious medical condition that could interfere with the study.
Patients who have had a recent surgery or are planning to have surgery soon.
Patients who are currently participating in another clinical trial.
Patients who have allergies to any of the substances used in the study.
Patients who are unable to follow the study procedures or attend the required visits.
Patients who are pregnant or breastfeeding.
Patients with a history of drug or alcohol abuse.
Patients with an infection that could affect the study results.
Patients who have a condition that affects their immune system, which is the body's defense against illness.
Patients who have a bleeding disorder, which means their blood does not clot properly.

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Responsive
Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert, Belgium

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Investigated drugs

NVD-003 is an autologous osteogenic bone graft used in the treatment of congenital pseudarthrosis of the tibia in pediatric patients. This means it is a bone graft made from the patient's own cells, specifically designed to help heal and strengthen the tibia, a bone in the lower leg. The goal of using NVD-003 in this trial is to evaluate its safety and effectiveness in promoting bone healing and growth in children with this condition. The study will also assess the safety of the procedures involved, including the collection of adipose tissue (fat tissue) from the patient and the surgery to implant the bone graft.

Autologous Adipose-Derived Stem Cells

What is already known about the treatment

NVD-003 – This medication is administered as an autologous osteogenic bone graft, meaning it is derived from the patient's own cells and used to promote bone growth. It is currently being studied in clinical trials for its safety and effectiveness in treating congenital pseudarthrosis of the tibia, a rare bone condition in children. The main therapeutic indication is to aid in bone healing and regeneration. 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 pharmacologically as a regenerative medicine product.

Investigated diseases

Congenital pseudarthrosis of the tibia – This is a rare condition where a child's tibia, or shinbone, does not form properly, leading to a false joint or nonunion. It is present at birth and can cause the affected leg to be weaker and more prone to fractures. Over time, the bone may not heal correctly, resulting in a persistent gap or instability in the bone. This condition can lead to difficulties in walking and may cause noticeable differences in leg length. The progression of the disease can vary, with some children experiencing worsening symptoms as they grow.