Congenital Pseudarthrosis of TibiaPhase 1NCT05693558
NVD-003 in the Treatment of Congenital Pseudarthrosis of the Tibia
A Phase 1 study testing NVD-003, an autologous 3D scaffold free osteogenic graft in people with Congenital Pseudarthrosis of Tibia, run by Novadip Biosciences at 2 sites in 2 countries.
Trial summary
A single arm, multi-country, multi-center study in pediatric patients, suffering from congenital pseudarthrosis of the tibia (CPT), treated during the primary surgical intervention with NVD-003, an autologous 3D scaffold free osteogenic graft. Read more about this study
- Who is it for?
- Ages 2 to 8 years
- Conditions the study is for
- Congenital Pseudarthrosis of Tibia
- What is being tested?
- NVD-003, an autologous 3D scaffold free osteogenic graft
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 24 Nov 2022to Jan 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 8 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosed with congenital pseudarthrosis of the tibia (with or without NF1) and presenting with a non-healing Paley type 3 or 4 fracture.
- Minimum weight of 5kg/11lbs.
- Maximum 2 previous failed surgical orthopaedic interventions to treat the primary CPT fracture.
- Acceptable serology and molecular test results excluding the presence of viruses
- Satisfactory general health condition to undergo surgeries (ATC and GS) with anaesthesia as per local standards.
- The patient's parent(s) and/or legal guardian(s) provided written informed consent and accepted the participation of the patient in the trial.
Who cannot join
- Bilateral CPT.
- Presence of CPT without a fracture of the tibia (Paley type 1 and 2).
- More than 2 failed surgical attempt(s) to treat the primary tibial fracture.
- Evidence of plexiform neurofibroma of any size or nodular fibroma ≥ 1.2in/3cm on the ipsilateral leg.
- Clinically significant infection at the target grafting site or systemic infection.
- Presence of clinically significant hematologic, renal, hepatic, and coagulation laboratory abnormalities (i.e., CBC, PT/INR, Chem-7, and LFTs, etc.).
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- Presence of any auto-immune disease, with exception of well controlled diabetes type 1 or auto-immune thyroid disorders.
- Any history of experimental therapy with another investigational drug within 60 days prior to screening.
- Presence of active tumour.
- Documented metabolic bone disease or any disorder, such as but not limited to osteogenesis imperfecta and osteomalacia, that could interfere with the bone healing and bone metabolism.
- Chronic, ongoing, or planned use of medications that might affect bone metabolism or bone quality such as bisphosphonates, steroids, methotrexate, anticoagulant therapies, immunosuppressant therapy or immunotherapy.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
Adult/Pediatric Limb Lengthening and Reconstruction - International Center for Limb Lengthening - Rubin Institute for Advanced Orthopedics Baltimore
Baltimore, Maryland, 21215
About this study
Official title: 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.
A single arm, multi-country, multi-center study in pediatric patients, suffering from congenital pseudarthrosis of the tibia (CPT), treated during the primary surgical intervention with NVD-003, an autologous 3D scaffold free osteogenic graft.
Read the detailed description
Congenital pseudarthrosis is a rare disorder of unknown aetiology and variable history that manifests itself as a non-union or pseudarthrosis of fractures that develop spontaneously or following minor trauma. It can be defined as a disorder of the diaphysis which is revealed by either pseudarthrosis at birth or by a pathological fracture presenting in bone with modifications such as bowing, narrowing of the medullary canal or a cyst. Although uncommon, CPT is the most frequently observed type of congenital pseudarthrosis. Its incidence is reported to be between 1:140,000 to 1:250,000 live births.
Autologous bone grafting is considered the gold standard approach as this material vascularizes and integrates with surrounding bone, minimizing the risk of infection, dislodgement, or break-down.
NVD-003 is a scaffold free 3D osteogenic graft derived from autologous adipose stem cells which become embedded in their extracellular matrix and combined with hydroxyapatite/beta-tricalcium phosphate (HA/βTCP) particles.
NVD-003 is intended to promote bone formation, supporting the physiological bone healing process in severe pathophysiological conditions such as hypoxia, lack of mineralized callus formation, bone resorption and low osteogenicity.
- Conditions
- Congenital Pseudarthrosis of Tibia
- Being tested
- NVD-003, an autologous 3D scaffold free osteogenic graft
- Keywords
- Tissue Engineered Therapy, bone graft, autologous, pediatric, congenital, non-union, pseudarthrosis, Cellular therapy
- Medical subject headings
- Pseudarthrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NVD-003, an autologous 3D scaffold free osteogenic graftBiological
Bone correction and grafting surgery
| Study group | Role | What they receive |
|---|---|---|
| NVD-003 bone graft implant | Experimental | The study includes 2 important surgical procedures, the Adipose Tissue Collection (ATC) and the Grafting Surgery (GS) and 3 stages
* Stage 1: A screening, adipose tissue collection \& NVD 003 manufacturing period.
* Stage 2: Grafting surgery and 12-month post-GS follow-up period.
* Stage 3: long-term safety follow-up period (from post-month 12 to month 24). Receives: Biological: NVD-003, an autologous 3D scaffold free osteogenic graft |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary objective: safety short (≤3 months) and mid long-term (>3-12 months): : Descriptive analysis
Assess number of patients with NVD-003 related (S)AEs
Measured: Up to 12 months post-grafting surgery
Secondary outcomes (5)
Long term Safety long-term (>12-24 months): Descriptive analysis
Assess number of patients with NVD-003 related SAEs
Measured: 12-24 months post grafting surgery
Tibial length evaluation
Assess evolution tibial length (using CT-Scan)
Measured: 3-, 6-, 12- and 24-months post-GS
Bone formation
Assess changes in bone formation (Using Lane and Sandhu Scoring on CT-Scan)
Measured: 3-, 6-, 12- and 24-months post-GS
Bone remodeling
Assess changes in bone remodeling (using Lane and Sandhu Scoring on CT-scan)
Based on Dual Energy CT-scan (DECT or Spectral CT), information of low- and high-energy photons is collected, allowing to uncover differences in energy-dependent attenuation, facilitating composition of NVD 003 and surrounding tissue and define bone mineral density) at 3-, 6-, and 12-months post-GS
Measured: 3-, 6-, 12- and 24-months post-GS
Bone union
Assess changes in bone union (using Lane and Sandhu Scoring on CT-scan)
Measured: 3-, 6-, 12- and 24-months post-GS
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2023
- Study start24 Nov 2022
- Primary completionJan 2025 (estimated)
- Study completionJan 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Congenital Pseudarthrosis of TibiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A single arm, multi-country, multi-center study in pediatric patients, suffering from congenital pseudarthrosis of the tibia (CPT), treated during the primary surgical intervention with NVD-003, an autologous 3D scaffold free osteogenic graft.
- Masking
- None (open label)
- Enrolment
- 4 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novadip Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Philip McClure, MD, Principal investigator, International Center for Limb Lengthening BaltimorePierre-Louis Docquier, Principal investigator, UCL St.Luc Brussels
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05693558
- Sponsor's protocol ID
- NVD003-CLN02 CPT
- Other IDs
- 2022-001282-12
- Status last verified
- Jan 2024
- First submitted
- 26 Oct 2022
- Last updated
- 26 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Paley D. Congenital pseudarthrosis of the tibia: biological and biomechanical considerations to achieve union and prevent refracture. J Child Orthop. 2019 Apr 1;13(2):120-133. doi: 10.1302/1863-2548.13.180147.
PubMed 30996736Dufrane D. Impact of Age on Human Adipose Stem Cells for Bone Tissue Engineering. Cell Transplant. 2017 Sep;26(9):1496-1504. doi: 10.1177/0963689717721203.
PubMed 29113460
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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.
- ClinicalTrials.gov ID
- NCT05693558
- Acronym / sponsor ID
- NVD003-CLN02 CPT
- Conditions
- Congenital Pseudarthrosis of Tibia
- Keywords
- Tissue Engineered Therapy, bone graft, autologous, pediatric, congenital, non-union, pseudarthrosis, Cellular therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 4 participants (estimated)
- Lead sponsor
- Novadip BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 24 Nov 2022 (actual)
- Primary completion
- Jan 2025 (estimated)
- Study completion
- Jan 2026 (estimated)
- First posted
- 23 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A single arm, multi-country, multi-center study in pediatric patients, suffering from congenital pseudarthrosis of the tibia (CPT), treated during the primary surgical intervention with NVD-003, an autologous 3D scaffold free osteogenic graft.
Detailed description
Congenital pseudarthrosis is a rare disorder of unknown aetiology and variable history that manifests itself as a non-union or pseudarthrosis of fractures that develop spontaneously or following minor trauma. It can be defined as a disorder of the diaphysis which is revealed by either pseudarthrosis at birth or by a pathological fracture presenting in bone with modifications such as bowing, narrowing of the medullary canal or a cyst. Although uncommon, CPT is the most frequently observed type of congenital pseudarthrosis. Its incidence is reported to be between 1:140,000 to 1:250,000 live births.
Autologous bone grafting is considered the gold standard approach as this material vascularizes and integrates with surrounding bone, minimizing the risk of infection, dislodgement, or break-down.
NVD-003 is a scaffold free 3D osteogenic graft derived from autologous adipose stem cells which become embedded in their extracellular matrix and combined with hydroxyapatite/beta-tricalcium phosphate (HA/βTCP) particles.
NVD-003 is intended to promote bone formation, supporting the physiological bone healing process in severe pathophysiological conditions such as hypoxia, lack of mineralized callus formation, bone resorption and low osteogenicity.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A single arm, multi-country, multi-center study in pediatric patients, suffering from congenital pseudarthrosis of the tibia (CPT), treated during the primary surgical intervention with NVD-003, an autologous 3D scaffold free osteogenic graft.
| Arm | Type | Receives |
|---|---|---|
| NVD-003 bone graft implant | Experimental | Biological: NVD-003, an autologous 3D scaffold free osteogenic graftThe study includes 2 important surgical procedures, the Adipose Tissue Collection (ATC) and the Grafting Surgery (GS) and 3 stages * Stage 1: A screening, adipose tissue collection \& NVD 003 manufacturing period. * Stage 2: Grafting surgery and 12-month post-GS follow-up period. * Stage 3: long-term safety follow-up period (from post-month 12 to month 24). |
Who may qualify
- Ages
- 2 to 8 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Diagnosed with congenital pseudarthrosis of the tibia (with or without NF1) and presenting with a non-healing Paley type 3 or 4 fracture.
- Minimum weight of 5kg/11lbs.
- Maximum 2 previous failed surgical orthopaedic interventions to treat the primary CPT fracture.
- Acceptable serology and molecular test results excluding the presence of viruses
- Satisfactory general health condition to undergo surgeries (ATC and GS) with anaesthesia as per local standards.
Key exclusion criteria (11 listed)
- Bilateral CPT.
- Presence of CPT without a fracture of the tibia (Paley type 1 and 2).
- More than 2 failed surgical attempt(s) to treat the primary tibial fracture.
- Evidence of plexiform neurofibroma of any size or nodular fibroma ≥ 1.2in/3cm on the ipsilateral leg.
- Clinically significant infection at the target grafting site or systemic infection.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NVD-003, an autologous 3D scaffold free osteogenic graftBone correction and grafting surgery | Biological | NVD-003 bone graft implant |
Primary outcome
Primary objective: safety short (≤3 months) and mid long-term (>3-12 months): : Descriptive analysis
Time frame: Up to 12 months post-grafting surgery
Assess number of patients with NVD-003 related (S)AEs
5 secondary outcomes
Long term Safety long-term (>12-24 months): Descriptive analysis
Time frame: 12-24 months post grafting surgery
Tibial length evaluation
Time frame: 3-, 6-, 12- and 24-months post-GS
Bone formation
Time frame: 3-, 6-, 12- and 24-months post-GS
Bone remodeling
Time frame: 3-, 6-, 12- and 24-months post-GS
Bone union
Time frame: 3-, 6-, 12- and 24-months post-GS
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jan 2024.
- Background: PubMed 30996736Paley D. Congenital pseudarthrosis of the tibia: biological and biomechanical considerations to achieve union and prevent refracture. J Child Orthop. 2019 Apr 1;13(2):120-133. doi: 10.1302/1863-2548.13.180147.
- Background: PubMed 29113460Dufrane D. Impact of Age on Human Adipose Stem Cells for Bone Tissue Engineering. Cell Transplant. 2017 Sep;26(9):1496-1504. doi: 10.1177/0963689717721203.
- Individual participant dataSharing planned: No
Related trials for Congenital Pseudarthrosis of Tibia
RecruitingPhase 3
A Study to Assess How Effective and Safe NVD003 is for Treating Patients With Congenital Pseudarthrosis of the Tibia.
Congenital Pseudarthrosis of Tibia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Philip McClure, MD principal investigator
International Center for Limb Lengthening Baltimore
Pierre-Louis Docquier principal investigator
UCL St.Luc Brussels
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing NVD-003, an autologous 3D scaffold free osteogenic graft in people with Congenital Pseudarthrosis of Tibia, run by Novadip Biosciences at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 26 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary objective: safety short (≤3 months) and mid long-term (>3-12 months): : Descriptive analysis (time frame: Up to 12 months post-grafting surgery). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05693558, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jan 2024.