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

Congenital pseudarthrosis of the tibia

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.

The following endpoints will be evaluated at 12 months after surgery with NVD003: • Radiographic healing • Clinical healing • Non-use of secondary interventions to promote or accelerate bone healing., • Number and severity of AEs related to NVD003 • Number of serious adverse events (SAEs) related to NVD003 • Units of blood transfused perioperatively (from start of surgical procedure until hospital discharge) • Duration of GS (in hours) • Duration of hospitalization after GS (in days) • Recurrent fracture within 12 months after GS., • Radiological bone union status at 3, 6, and 12 months after GS, assessed centrally by ICRs using the total extended Lane and Sandhu scoring (eLSS) method • Functional walking outcome, assessed using the timed 10-meter walking test (when appropriate) post GS at 3, 6, and 12 months., • Mean change from Baseline to 6 and 12 months after GS in QoL: o Pediatric Outcomes Data Collection Instrument (PODCI) parent report for children 2 years of age and above.

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