A Study to Assess How Effective and Safe NVD003 is for Treating Patients With Congenital Pseudarthrosis of the Tibia.

NCT07112443 | Recruiting Phase 3 FDA Drug

• 2 other identifiers: NVD003-CLN032025-520998-39-00
• Study Type: interventional
• Target: 12
• Locations: 4 countries
• Sites: 7

Brief 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.

Conditions

Congenital Pseudarthrosis of Tibia

Keywords

tissue engineered therapy; bone graft; autologous; pediatric; congenital; non-union; pseudarthrosis; cellular therapy

Outcome Measures

Primary Outcomes (1)

• 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.

  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

  12 months after grafting surgery

• Efficacy: To assess the efficacy of NVD003 with respect to individual components of overall healing - clinical healing

  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

  12 months after grafting surgery

• Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - Adverse events

  12 months after grafting surgery

• Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants - Serious adverse events

  12 months after grafting surgery

Study Arms (2)

Experimental Arm

EXPERIMENTAL

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

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.

Procedure: Iliac Crest Bone Graft

Interventions

NVD003 BIOLOGICAL

Bone correction and grafting surgery

Experimental Arm

Iliac Crest Bone Graft PROCEDURE

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.

Standard of Care Arm

Eligibility Criteria

• Age: Up to 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• 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:

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Novadip Biosciences: lead

Study Sites (7)

Phoenix Children's Hospital, Inc.

Phoenix, Arizona, 85016, United States

RECRUITING

Loma Linda University Health

Loma Linda, California, 92354, United States

RECRUITING

LifeBridge Health - International Center for Limb Lengthening

Baltimore, Maryland, 21117, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

Cliniques Universitaires Saint-Luc ASBL (CUSCL)

Brussels, Brussels Capital, 1200, Belgium

RECRUITING

CHU Amiens-Picardie

Amiens, Picardie, 80000, France

RECRUITING

Hospital Sant Joan de Déu

Esplugues de Llobregat, 08950, Spain

RECRUITING

MeSH Terms

Conditions

Pseudarthrosis

Condition Hierarchy (Ancestors)

Fractures, Ununited; Fractures, Bone; Wounds and Injuries

Central Study Contacts

Judy Ashworth, MD

CONTACT

224-713-6154; clinical@novadip.com

Lola TOME

CONTACT

+447468479913; regulatory@novadip.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 23, 2025
• First Posted: August 8, 2025
• Study Start: September 2, 2025
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): July 1, 2027
• Last Updated: April 1, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (4); FR (1); BE (1); ES (1)