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.
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 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).
Parallel
NVD003
Biological/VaccineBone correction and grafting surgery
Iliac Crest Bone Graft
Procedure/SurgeryEither 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.
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.
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)
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
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
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
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