NVD003 for Tibial Pseudoarthrosis

Overseen By Judy Ashworth, MD

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Novadip Biosciences

Must not be taking: Bisphosphonates, Steroids, Methotrexate, others

Disqualifiers: Bilateral CPT, Autoimmune disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

12 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of a new treatment called NVD003 for children with Congenital Pseudarthrosis of the Tibia (CPT), a condition affecting the bone in one leg that hinders healing. Researchers will compare NVD003, an experimental treatment, with the standard treatment, iliac crest bone graft (taking bone from the hip), to determine which is more effective for bone healing after surgery. Children with CPT that isn't healing well and who are ready for surgery using internal rods might be suitable candidates for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot use medications that might affect bone metabolism or quality, like steroids or methotrexate, during the study.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that NVD003 is generally safe. In an earlier study, 17 people, including both adults and children, received NVD003. The study found it effective for 88% of participants without major safety issues. Over five years, NVD003 integrated well with bone and did not degrade, aiding in bone healing and strength.

Other studies confirm that NVD003 is safe and effective for promoting quick and lasting bone healing. These findings suggest that NVD003 is usually well-tolerated, making it a promising option for those considering participation in a clinical trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for tibial pseudoarthrosis, which often involve traditional bone grafting techniques like the iliac crest bone graft, NVD003 introduces a new surgical approach. This treatment is unique because it uses internal fixation with cross-union specifically designed for enhanced stability and bone healing. Researchers are excited about NVD003 because it potentially offers a more robust and precise method to encourage bone growth and healing, which could lead to better recovery outcomes for patients. This new approach might significantly reduce healing time and improve the overall success rate compared to conventional methods.

What evidence suggests that this trial's treatments could be effective for tibial pseudoarthrosis?

Studies have shown promising results for NVD003, a treatment designed to heal bone problems. In one study, 89% of patients who received NVD003 experienced successful bone healing without repeat fractures after two years. Another study found that 88% of participants, including both adults and children, experienced positive results with NVD003. The treatment demonstrated stable and lasting bone healing, particularly in challenging cases where bones would not heal independently. In this trial, participants will be assigned to either the Experimental Arm, receiving NVD003, or the Standard of Care Arm, which involves standard surgical grafting procedures. These findings suggest that NVD003 could be an effective option for treating congenital pseudarthrosis of the tibia in children. ^{1 2 3 5 6}

Are You a Good Fit for This Trial?

This trial is for children under 17 with Congenital Pseudarthrosis of the Tibia (CPT), specifically those with non-healing fractures suitable for internal fixation surgery. They must weigh at least 11 pounds, not have certain viruses or syphilis, and aren't pregnant. Consent from parents or guardians is required.

Inclusion Criteria

I weigh at least 11 pounds.

My parent or guardian has agreed to my participation in the study.

I am 17 years old or younger.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Surgical Treatment

Participants undergo surgical grafting procedure with internal fixation using either NVD003 or iliac crest bone graft

Day 1

1 visit (in-person)

Post-Surgical Follow-up

Participants are monitored for safety and efficacy, including assessments of clinical and radiographic healing, and adverse events

12 months

Regular visits at 3, 6, and 12 months

Long-term Follow-up

Participants are monitored for long-term outcomes such as recurrent fracture and quality of life

Beyond 12 months

What Are the Treatments Tested in This Trial?

Interventions

• Iliac Crest Bone Graft
• NVD003

Trial Overview The study compares NVD003 treatment to traditional iliac crest bone grafts in healing tibial pseudoarthrosis in kids. It aims to see which method is safer and more effective after one year post-surgery.

How Is the Trial Designed?

Group I: Experimental Arm Experimental Treatment 1 Intervention

surgical grafting procedure: internal fixation with cross-union using NVD003

Group II: Standard of Care Arm Active Control 1 Intervention

Either a single-stage grafting surgery with internal fixation and cross-union or the 2-stage "induced membrane" grafting surgery approach, depending on the surgeon's own standard of Care.

Who Is Running the Clinical Trial?

Novadip Biosciences

Lead Sponsor

Trials

6

Recruited

90+

Citations

1. newscienceventures.com newscienceventures.com/novadip-releases-five-year-follow-up-results-for-nvd003-regenerative-treatment-for-severe-bone-non-union-of-lower-limb/

Novadip releases five-year follow-up results for NVD003, ... At 24-months post-implantation of NVD003, eight of the nine patients (89%) had achieved bone union and no refractures have been reported during ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT07112443

A Study to Assess How Effective and Safe NVD003 is ... The purpose of this study is to assess the efficacy and safety of NVD003 in pediatric participants with unilateral Congenital Pseudarthrosis of the Tibia (CPT) ...

3. novadip.com novadip.com/press-releases/novadip-reports-12-month-results-from-phase-1b-2a-trial-of-nvd003-to-treat-rare-pediatric-disease-congenital-pseudarthrosis-of-the-tibia/

Novadip reports 12-month results from phase 1b/2a trial of ... 88% efficacy demonstrated across all patients (n=17) treated with. NVD003 to date (nine adults with bone non-union and eight children

4. firstwordpharma.com firstwordpharma.com/story/5684207

Novadip Biosciences SA Announces Positive Results from ... Two-year clinical and radiological follow up confirmed NVD-003 achieved stable and irreversible bone healing in patients with recalcitrant bone ...

5. ala.associates ala.associates/regulatory/novadip-receives-rmat-designation-for-nvd003-its-regenerative-treatment-for-congenital-pseudarthosis-of-the-tibia/

Novadip receives RMAT designation for NVD003, its ... “NVD003 perfectly matches the FDA requirements for this designation, as it is a tissue regeneration product intended as a treatment for CPT, a ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12470636/

An Autologous Human Adipose Stem Cell-Derived 3D ... NVD003 facilitated bone union in adult and pediatric patients even under severe pathophysiological conditions. Keywords: autograft, bone ...