Congenital Pseudarthrosis of TibiaPhase 3NCT07112443
A Study to Assess How Effective and Safe NVD003 is for Treating Patients With Congenital Pseudarthrosis of the Tibia.
A Phase 3 study testing NVD003 and Iliac Crest Bone Graft in people with Congenital Pseudarthrosis of Tibia, run by Novadip Biosciences at 7 sites in 4 countries.
Trial summary
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) compared with iliac crest bone graft (ICBG) at 12 months post graft surgery. Read more about this study
- Who is it for?
- Ages Up to 17 years
- Conditions the study is for
- Congenital Pseudarthrosis of Tibia
- What is being tested?
- NVD003 and Iliac Crest Bone Graft
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 7 sitesin 4 countries
- Study dates
- 2 Sep 2025to Jul 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 Up to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participant's parent(s)/legal guardian(s) have provided written informed consent (and assent has been provided by the participant, depending on age) for the study.
- Participant is of any sex, ≤17 years of age.
- Participant has been diagnosed with CPT (with or without NF1).
- Participant has a non-healing Paley type 3 or 4 diaphyseal fracture.4
- Participant is a candidate for surgical treatment using an internal fixation approach (intramedullary rod) based on CPT fracture status and general health status.
- Participant has serology and molecular test results at Visits 1 and 2 excluding the presence of human T-cell lymphoma virus, human immunodeficiency virus, hepatitis B virus, hepatitis C virus, and syphilis.
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- Participant can provide an adequate ATC sample volume.
- Participant weighs ≥5 kg/11 lb at Screening and on Day 1.
- Participant is not pregnant or lactating.
- If participant is of childbearing potential, is practicing highly effective methods of birth control from Screening to the end of the study:
- Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
- Oral
- Intravaginal
- Transdermal
- Progestogen-only hormonal contraception associated with inhibition of ovulation:
- Oral
- Injectable
- Implantable
- Intrauterine device
- Intrauterine hormone-releasing system
- Bilateral tubal occlusion
- Vasectomized partner
- Sexual abstinence, defined as refraining from heterosexual intercourse during study participation, is acceptable if this is the participant's usual lifestyle; periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and the lactational amenorrhea method are not acceptable methods of contraception Note: A participant is considered to be of childbearing potential if they are postmenarchal and premenopausal, unless surgically sterile (permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy).
- If participant is sexually active and has a partner who may become pregnant (i.e., neither surgically sterile nor postmenopausal), agrees to use highly effective contraception (e.g., sterilization, birth control pills, Depo Provera injections, or contraceptive implants) from Screening to the end of the study.
- Participant agrees to refrain from donating sperm or eggs from Screening to the end of the study.
- Participant and parent(s)/legal guardian(s) are able to understand all study information provided and are willing to return to the study facility for all visits, including follow-up evaluations.
Who cannot join
- Participant has bilateral CPT.
- Participant has evidence of plexiform neurofibroma of any size or nodular fibroma ≥1.2 inches/3 cm on the ipsilateral leg.
- Participant has a clinically significant infection at the fracture site or systemic infection.
- Participant's CPT fracture involves the metaphysis (i.e., not limited to the diaphysis).
- Participant has a CPT fracture, for which the surgeon intends to use an external fixation system (e.g., Ilizarov, Taylor spatial frame, rail, etc.) instead of, or in addition to, internal fixation.
- Participant has an autoimmune disease, with the exception of well-controlled type 1 diabetes or autoimmune thyroid disorders.
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- Participant has an active (malignant) tumor.
- Participant has documented metabolic bone disease or any disorder, such as, but not limited, to osteogenesis imperfecta and osteomalacia, that could interfere with bone healing and bone metabolism.
- Participant has any chronic, ongoing, or planned use of medications that might affect bone metabolism or bone quality such as bisphosphonates, steroids, methotrexate, vitamin K antagonists, immunosuppressant therapy, or immunotherapy during the study.
- Note that perioperative treatment with a bisphosphonate is allowed in Cohort A participants randomized to ICBG if its use is deemed SOC by the treating surgeon.
- Participant has any history of allergic reaction or any anticipated hypersensitivity to any anesthetic agent or any potential hypersensitivity to any of the components of the NVD003 graft (including the CMRL1066 formulation medium) or hypersensitivity related to other factors in the surgical process for the ICBG graft, such as anesthesia, medications, suture materials or fixation devices.
- Participant has received any investigational product (including a device) within 60 days before enrollment in the study.
- Participant would be concurrently enrolled in another clinical study while participating in this study.
- Participant has any clinically significant hematologic, renal, hepatic, and coagulation laboratory abnormalities (i.e., complete blood count, prothrombin time/international normalized ratio, Chem-7, liver function tests, etc.).
- Participant or participant's parent(s)/legal guardian(s) have an unstable condition (e.g., psychiatric disorder, a recent history of substance abuse) or is otherwise thought to be unreliable or incapable of complying with the requirements of the protocol.
Trial locations and contacts
7 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 4 countries
United States4 sites, 4 recruiting
- Recruiting
LifeBridge Health - International Center for Limb Lengthening
Baltimore, Maryland, 21117
Philip McClure, MD
- Recruiting
Loma Linda University Health
Loma Linda, California, 92354
Corey Fuller, MD
- Recruiting
Phoenix Children's Hospital, Inc.
Phoenix, Arizona, 85016
Mohan Belthur, MD
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
Noelle A Larson, MD
Belgium1 site, 1 recruiting
- Recruiting
Cliniques Universitaires Saint-Luc ASBL (CUSCL)
Brussels, Brussels Capital, 1200
Pierre-Louis Docquier, Pr.
France1 site, 1 recruiting
- Recruiting
CHU Amiens-Picardie
Amiens, Picardie, 80000
Céline Klein, Pr.
Spain1 site, 1 recruiting
- Recruiting
Hospital Sant Joan de Déu
Esplugues de Llobregat, 08950
CÉSAR GALO GARCÍA FONTECHA, DR.
About this study
Official title: A Phase 3 Randomized, Controlled Study to Assess the Efficacy and Safety of NVD003 Compared With Iliac Crest Bone Graft in Pediatric Participants Treated Surgically for Congenital Pseudarthrosis of the Tibia
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) compared with iliac crest bone graft (ICBG) at 12 months post graft surgery.
Read the detailed description
CPT is a rare disorder affecting the tibia and/or the ipsilateral fibula in children. The disease is usually diagnosed during the first year of life and is characterized by nonunion of a tibial diaphyseal fracture that either develops spontaneously or after a history of trivial trauma in a previously dysplastic segment of the bone. The treatment of CPT remains a challenge, often due to failure of ICBG to achieve and maintain bone union, resulting in multiple revision surgeries and occasionally amputation of the lower leg. NVD003 is a novel 3-dimensional osteogenic graft that is derived from autologous adipose stem cells combined with hydroxyapatite/beta-tricalcium phosphate particles. As an alternative to ICBG, NVD003 is intended to promote bone formation and support the bone healing process, even in severely disturbed pathophysiological conditions like CPT.
This is a prospective, randomized, controlled, multicenter, phase 3 clinical study in pediatric participants with CPT who have a non-healing Paley type 3 or 4 diaphyseal fracture and are candidates to undergo surgical treatment with internal fixation (intramedullary rod).
- Conditions
- Congenital Pseudarthrosis of Tibia
- Being tested
- NVD003, Iliac Crest Bone 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.
NVD003Biological
Bone correction and grafting surgery
Iliac Crest Bone GraftProcedure or surgery
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.
| Study group | Role | What they receive |
|---|---|---|
| Experimental Arm | Experimental | surgical grafting procedure: internal fixation with cross-union using NVD003 Receives: Biological: NVD003 |
| Standard of Care Arm | Active comparator | 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. Receives: Procedure: Iliac Crest Bone Graft |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the efficacy of NVD003 for the surgical treatment of CPT in pediatric participants
Overall healing at 12 months, defined as a binary (yes/no) outcome derived from the following 3 outcomes:
- Radiographic healing, defined as a score ≥13 on the modified Radiological Union Scale for Tibia (mRUST) scale (range 1 to 16) assessed by independent central readers (ICRs)
- Clinical healing measured by the absence of pain with weight-bearing, based on clinician's assessment
- No use of secondary interventions to promote or accelerate bone healing All 3 criteria must be met for a "yes" on overall healing for this endpoint to be achieved.
Measured: At 12 months post GS
Secondary outcomes (12)
Efficacy: To assess the efficacy of NVD003 with respect to individual components of overall healing - radiographic healing
Radiographic healing, defined as a score ≥13 on the modified Radiological Union Scale for Tibia (mRUST) scale (range 1 to 16) assessed by independent central readers (ICRs)
Measured: 12 months after grafting surgery
Efficacy: To assess the efficacy of NVD003 with respect to individual components of overall healing - clinical healing
Clinical healing measured by the absence of pain with weight-bearing, based on clinician's assessment
Measured: 12 months after grafting surgery
Efficacy: To assess the efficacy of NVD003 with respect to individual components of overall healing - No use of secondary intervention
No use of secondary interventions to promote or accelerate bone healing
Measured: 12 months after grafting surgery
Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - Adverse events
Number and severity of adverse events (AEs) related to NVD003
Measured: 12 months after grafting surgery
Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - Serious adverse events
Number of serious adverse events (SAEs) related to NVD003
Measured: 12 months after grafting surgery
Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - Units of blood transfused perioperatively
Units of blood transfused perioperatively (in Liters or milliliters)
Measured: from start of surgical procedure (Day 1) until hospital discharge
Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - Duration of Grafting Surgery
Duration of Grafting Surgery (in hours)
Measured: Grafting Surgery (Day 1)
Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - duration of hospitalization
Duration of hospitalization after GS (in days)
Measured: from grafting surgery (Day 1) until hospital discharge
Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - Recurrent fracture
Recurrent fracture within 12 months after GS
Measured: 12 months after grafting surgery
To further evaluate the efficacy of NVD003 for the surgical treatment of CPT in pediatric participants based on other measures of bone healing - Radiological
- Radiological bone union status at 3, 6, and 12 months after Grafting Surgery, assessed centrally by independent central readers (ICRs) using the total extended Lane and Sandhu scoring (eLSS) method (maximum of 12 points)
Measured: Post grafting surgery (Day 1), and at months 3, 6 and 12
To further evaluate the efficacy of NVD003 for the surgical treatment of CPT in pediatric participants based on other measures of bone healing - functional walking
Functional walking outcome, assessed using the timed 10-meter walking test (when appropriate) post Grafting Surgery at 3, 6, and 12 months (in second)
Measured: Post grafting surgery (Day 1), and at months 3, 6 and 12
To evaluate the effects of NVD003 on quality of life (QoL)
Mean change from Baseline to 6 and 12 months after GS in QoL:
- Pediatric Outcomes Data Collection Instrument (PODCI) parent report for children 2 years of age and above. ("0" represents a poor outcome/worse health while "100" is the best possible outcome/best health.)
Measured: Baseline and months 6 and 12 post grafting surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2025
- Study start2 Sep 2025
- Primary completionOct 2026 (estimated)
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novadip Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07112443
- Sponsor's protocol ID
- NVD003-CLN03
- Other IDs
- 2025-520998-39-00, 2025
- Status last verified
- Mar 2026
- First submitted
- 23 Jul 2025
- Last updated
- 1 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Randomized, Controlled Study to Assess the Efficacy and Safety of NVD003 Compared With Iliac Crest Bone Graft in Pediatric Participants Treated Surgically for Congenital Pseudarthrosis of the Tibia
- ClinicalTrials.gov ID
- NCT07112443
- Acronym / sponsor ID
- NVD003-CLN03
- Conditions
- Congenital Pseudarthrosis of Tibia
- Keywords
- tissue engineered therapy, bone graft, autologous, pediatric, congenital, non-union, pseudarthrosis, cellular therapy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Novadip BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 2 Sep 2025 (actual)
- Primary completion
- Oct 2026 (estimated)
- Study completion
- Jul 2027 (estimated)
- First posted
- 8 Aug 2025 (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.
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) compared with iliac crest bone graft (ICBG) at 12 months post graft surgery.
Detailed description
CPT is a rare disorder affecting the tibia and/or the ipsilateral fibula in children. The disease is usually diagnosed during the first year of life and is characterized by nonunion of a tibial diaphyseal fracture that either develops spontaneously or after a history of trivial trauma in a previously dysplastic segment of the bone. The treatment of CPT remains a challenge, often due to failure of ICBG to achieve and maintain bone union, resulting in multiple revision surgeries and occasionally amputation of the lower leg. NVD003 is a novel 3-dimensional osteogenic graft that is derived from autologous adipose stem cells combined with hydroxyapatite/beta-tricalcium phosphate particles. As an alternative to ICBG, NVD003 is intended to promote bone formation and support the bone healing process, even in severely disturbed pathophysiological conditions like CPT.
This is a prospective, randomized, controlled, multicenter, phase 3 clinical study in pediatric participants with CPT who have a non-healing Paley type 3 or 4 diaphyseal fracture and are candidates to undergo surgical treatment with internal fixation (intramedullary rod).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Experimental Arm | Experimental | Biological: NVD003surgical grafting procedure: internal fixation with cross-union using NVD003 |
| Standard of Care Arm | Active comparator | Procedure: Iliac Crest Bone GraftEither 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 may qualify
- Ages
- Up to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (26 listed)
- Participant's parent(s)/legal guardian(s) have provided written informed consent (and assent has been provided by the participant, depending on age) for the study.
- Participant is of any sex, ≤17 years of age.
- Participant has been diagnosed with CPT (with or without NF1).
- Participant has a non-healing Paley type 3 or 4 diaphyseal fracture.4
- Participant is a candidate for surgical treatment using an internal fixation approach (intramedullary rod) based on CPT fracture status and general health status.
Key exclusion criteria (15 listed)
- Participant has bilateral CPT.
- Participant has evidence of plexiform neurofibroma of any size or nodular fibroma ≥1.2 inches/3 cm on the ipsilateral leg.
- Participant has a clinically significant infection at the fracture site or systemic infection.
- Participant's CPT fracture involves the metaphysis (i.e., not limited to the diaphysis).
- Participant has a CPT fracture, for which the surgeon intends to use an external fixation system (e.g., Ilizarov, Taylor spatial frame, rail, etc.) instead of, or in addition to, internal fixation.
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 |
|---|---|---|
| NVD003Bone correction and grafting surgery | Biological | Experimental Arm |
| Iliac Crest Bone GraftEither 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. | Procedure or surgery | Standard of Care Arm |
Primary outcome
To assess the efficacy of NVD003 for the surgical treatment of CPT in pediatric participants
Time frame: At 12 months post GS
Overall healing at 12 months, defined as a binary (yes/no) outcome derived from the following 3 outcomes: * Radiographic healing, defined as a score ≥13 on the modified Radiological Union Scale for Tibia (mRUST) scale (range 1 to 16) assessed by independent central readers (ICRs) * Clinical healing measured by the absence of pain with weight-bearing, based on clinician's assessment * No use of secondary interventions to promote or accelerate bone healing All 3 criteria must be met for a "yes" on overall healing for this endpoint to be achieved.
12 secondary outcomes
Efficacy: To assess the efficacy of NVD003 with respect to individual components of overall healing - radiographic healing
Time frame: 12 months after grafting surgery
Efficacy: To assess the efficacy of NVD003 with respect to individual components of overall healing - clinical healing
Time frame: 12 months after grafting surgery
Efficacy: To assess the efficacy of NVD003 with respect to individual components of overall healing - No use of secondary intervention
Time frame: 12 months after grafting surgery
Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - Adverse events
Time frame: 12 months after grafting surgery
Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - Serious adverse events
Time frame: 12 months after grafting surgery
Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - Units of blood transfused perioperatively
Time frame: from start of surgical procedure (Day 1) until hospital discharge
Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - Duration of Grafting Surgery
Time frame: Grafting Surgery (Day 1)
Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - duration of hospitalization
Time frame: from grafting surgery (Day 1) until hospital discharge
Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - Recurrent fracture
Time frame: 12 months after grafting surgery
To further evaluate the efficacy of NVD003 for the surgical treatment of CPT in pediatric participants based on other measures of bone healing - Radiological
Time frame: Post grafting surgery (Day 1), and at months 3, 6 and 12
To further evaluate the efficacy of NVD003 for the surgical treatment of CPT in pediatric participants based on other measures of bone healing - functional walking
Time frame: Post grafting surgery (Day 1), and at months 3, 6 and 12
To evaluate the effects of NVD003 on quality of life (QoL)
Time frame: Baseline and months 6 and 12 post grafting surgery
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2026.
- Individual participant dataSharing planned: No
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.
7 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NVD003 and Iliac Crest Bone Graft in people with Congenital Pseudarthrosis of Tibia, run by Novadip Biosciences at 7 sites in 4 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 26 inclusion and 15 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: looking for participants now, as of the sponsor's last update on 1 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Belgium, France, Spain. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Judy Ashworth, MD (224-713-6154, clinical@novadip.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To assess the efficacy of NVD003 for the surgical treatment of CPT in pediatric participants (time frame: At 12 months post GS). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 1 Apr 2026.