NVD-003 in the Treatment of Congenital Pseudarthrosis of the Tibia

NCT05693558 | Unknown Phase 1 FDA Drug

• 2 other identifiers: NVD003-CLN02 CPT2022-001282-12
• Study Type: interventional
• Target: 4
• Locations: 2 countries
• Sites: 2

Brief Summary

A single arm, multi-country, multi-center study in pediatric patients, suffering from congenital pseudarthrosis of the tibia (CPT), treated during the primary surgical intervention with NVD-003, an autologous 3D scaffold free osteogenic graft.

Conditions

Congenital Pseudarthrosis of Tibia

Keywords

Tissue Engineered Therapy; bone graft; autologous; pediatric; congenital; non-union; pseudarthrosis; Cellular therapy

Outcome Measures

Primary Outcomes (1)

• Primary objective: safety short (≤3 months) and mid long-term (>3-12 months): : Descriptive analysis

  Assess number of patients with NVD-003 related (S)AEs

  Up to 12 months post-grafting surgery

Secondary Outcomes (5)

• Long term Safety long-term (>12-24 months): Descriptive analysis

  12-24 months post grafting surgery

• Tibial length evaluation

  3-, 6-, 12- and 24-months post-GS

• Bone formation

  3-, 6-, 12- and 24-months post-GS

• Bone remodeling

  3-, 6-, 12- and 24-months post-GS

• Bone union

  3-, 6-, 12- and 24-months post-GS

Study Arms (1)

NVD-003 bone graft implant

EXPERIMENTAL

The study includes 2 important surgical procedures, the Adipose Tissue Collection (ATC) and the Grafting Surgery (GS) and 3 stages * Stage 1: A screening, adipose tissue collection \& NVD 003 manufacturing period. * Stage 2: Grafting surgery and 12-month post-GS follow-up period. * Stage 3: long-term safety follow-up period (from post-month 12 to month 24).

Biological: NVD-003, an autologous 3D scaffold free osteogenic graft

Interventions

NVD-003, an autologous 3D scaffold free osteogenic graft BIOLOGICAL

Bone correction and grafting surgery

NVD-003 bone graft implant

Eligibility Criteria

• Age: 2 Years - 8 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosed with congenital pseudarthrosis of the tibia (with or without NF1) and presenting with a non-healing Paley type 3 or 4 fracture.
• Minimum weight of 5kg/11lbs.
• Maximum 2 previous failed surgical orthopaedic interventions to treat the primary CPT fracture.
• Acceptable serology and molecular test results excluding the presence of viruses
• Satisfactory general health condition to undergo surgeries (ATC and GS) with anaesthesia as per local standards.
• The patient's parent(s) and/or legal guardian(s) provided written informed consent and accepted the participation of the patient in the trial.

You may not qualify if:

• Bilateral CPT.
• 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.
• 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.
• Any history of experimental therapy with another investigational drug within 60 days prior to screening.
• Presence of active tumour.
• 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.

Sponsors & Collaborators

• Novadip Biosciences: lead

Study Sites (2)

Adult/Pediatric Limb Lengthening and Reconstruction - International Center for Limb Lengthening - Rubin Institute for Advanced Orthopedics Baltimore

Baltimore, Maryland, 21215, United States

Location

Cliniques Universitaires Saint-Luc

Brussels, 1200, Belgium

Location

Related Publications (2)

• Paley D. Congenital pseudarthrosis of the tibia: biological and biomechanical considerations to achieve union and prevent refracture. J Child Orthop. 2019 Apr 1;13(2):120-133. doi: 10.1302/1863-2548.13.180147.

  PMID: 30996736 BACKGROUND

• Dufrane D. Impact of Age on Human Adipose Stem Cells for Bone Tissue Engineering. Cell Transplant. 2017 Sep;26(9):1496-1504. doi: 10.1177/0963689717721203.

  PMID: 29113460 BACKGROUND

MeSH Terms

Conditions

Pseudarthrosis

Condition Hierarchy (Ancestors)

Fractures, Ununited; Fractures, Bone; Wounds and Injuries

Study Officials

• Philip McClure, MD

  International Center for Limb Lengthening Baltimore

  PRINCIPAL INVESTIGATOR

• Pierre-Louis Docquier

  UCL St.Luc Brussels

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: A single arm, multi-country, multi-center study in pediatric patients, suffering from congenital pseudarthrosis of the tibia (CPT), treated during the primary surgical intervention with NVD-003, an autologous 3D scaffold free osteogenic graft.

Study Record Dates

• First Submitted: October 26, 2022
• First Posted: January 23, 2023
• Study Start: November 24, 2022
• Primary Completion: January 1, 2025
• Study Completion: January 1, 2026
• Last Updated: January 26, 2024

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1); BE (1)