A Study to Assess How Effective and Safe NVD003 is for Treating Patients With Congenital Pseudart… (NCT07112443) | Clinical Trial Compass
RecruitingPhase 3
A Study to Assess How Effective and Safe NVD003 is for Treating Patients With Congenital Pseudarthrosis of the Tibia.
United States, Belgium12 participantsStarted 2025-09-02
Plain-language 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.
Who can participate
Age range17 Years
SexALL
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Inclusion criteria
✓. 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.
Exclusion criteria
✕. 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).
What they're measuring
1
To assess the efficacy of NVD003 for the surgical treatment of CPT in pediatric participants
✕. 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.