A proof of concept study with NVD-003, an autologous osteogenic bone graft, in the treatment of congenital pseudarthrosis of the tibia in pediatric patients.
- Conditions
- Congenital pseudarthrosis of the tibiaTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2024-515478-29-00
- Lead Sponsor
- ovadip Biosciences
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 4
Male or female pediatric patients: a.Diagnosed with congenital pseudarthrosis of the tibia (with or without NF1) and presenting with a non-healing Paley type 3 or 4 fracture. b.With a minimum weight of 5kg/11lbs. c.Within the age range of 2 years to 8 years., Maximum 2 previous failed surgical orthopedic interventions to treat the primary CPT fracture., Patient fulfills criteria for undergoing a surgical intervention (ATC and GS) as per standard of care., Patient has serology and molecular test results excluding the presence of Human T-cell lymphoma virus (HTLV1/2), human immunodeficiency virus (HIV 1/2), hepatitis B virus (HBV), hepatitis C virus (HCV), and syphilis, Possibility to obtain a minimum of 2cc/ 0.07fl.oz. (preferably up to 10cc/ 0.34fl oz) of adipose tissue., Satisfactory general health condition to undergo surgeries (ATC and GS) with anesthesia as per local standards., The fracture and patient baseline characteristics allow the application of a pre-defined standardized surgical procedure as detailed in the study specific procedures section 6.1.1.4., A NVD 003 graft volume need not exceeding 20cc (0.68fl oz), confirmed by the treating orthopedic surgeon based on radiological measurements. The estimated volume should cover the total of the residual tibial bone defect volume of 0.5 to 1cm (0.2 0.39in) in height, the cross-union tibial bone-fibula volume and the eventual fibular bone defect volume., The patient’s parent(s) and/or legal guardian(s) provided written informed consent and accepted the participation of the patient in the trial.
Bilateral CPT., Any history of experimental therapy with another investigational drug within 60 days prior to screening., Presence of active tumor., Documented metabolic bone disease or any disorder, such as but not limited to osteogenesis imperfecta and osteomalacia, that could interfere with the bone healing and bone metabolism., Chronic, ongoing, or planned use of medications that might affect bone metabolism or bone quality such as bisphosphonates, steroids, methotrexate, anticoagulant therapies, immunosuppressant therapy or immunotherapy., Patient was breastfed less than 1 year before the lipo-aspiration., Any condition which, in the opinion of the investigator, could interfere with trial conduct, the patient’s compliance with the protocol or influence interpretation of the results., Presence of CPT without a fracture of the tibia (Paley type 1 and 2)., More than 2 failed surgical attempt(s) to treat the primary tibial fracture., Evidence of plexiform neurofibroma of any size or nodular fibroma = 1.2in/3cm on the ipsilateral leg., Clinically significant infection at the target grafting site or systemic infection., History of allergic reaction or any anticipated hypersensitivity to any anesthetic agent or any potential hypersensitivity to any of the components of the NVD-003 graft (including the CMRL1066 transport medium)., CPT fracture requiring the use of one of the following surgical techniques: a.An external fixation system (e.g. Ilizarov, TSF, rail, …); b.A carrier or scaffold; c.An autogenous recombinant human bone morphogenetic protein., Presence of clinically significant hematologic, renal, hepatic, and coagulation laboratory abnormalities (i.e., CBC, PT/INR, Chem-7, and LFTs, etc.)., Presence of any auto-immune disease, with exception of well controlled diabetes type 1 or auto-immune thyroid disorders.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Safety: to locally assess clinical and radiological safety: assess short, mid long and long-term safety of the NVD-003 treatment (including the adipose tissue collection procedure and the NVD-003 grafting surgery);Secondary Objective: To centrally assess NVD 003 efficacy radiologically (bone formation, union and remodeling), To locally assess NVD 003 efficacy clinically;Primary end point(s): Short term safety focusses on ATC and NVD 003 related (S)AEs between screening and 3 months post-GS (V6 included), Mid long-term safety focusses on the NVD 003 graft implantation, NVD 003 related (S)AEs and the surgical reintervention rate, including all corrective surgeries following a confirmed failure of the index surgery, captured between 3 months (V6 excluded) and 12 months post-GS (V8 included), Long-term safety evaluates the NVD 003 related SAEs between 12 months post-GS (V8 excluded) and 24 months (V9 included)
- Secondary Outcome Measures
Name Time Method