RECRUITING

A Study to Assess How Effective and Safe NVD003 is for Treating Patients With Congenital Pseudarthrosis of the Tibia.

Conditions

Congenital Pseudarthrosis of Tibia tissue engineered therapy bone graft autologous pediatric congenital non-union pseudarthrosis cellular therapy

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2025-09-02

Study Completion:2027-07

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT07112443

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 17 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
1. 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. 2. Participant is of any sex, ≤17 years of age. 3. Participant has been diagnosed with CPT (with or without NF1). 4. Participant has a non-healing Paley type 3 or 4 diaphyseal fracture.4 5. Participant is a candidate for surgical treatment using an internal fixation approach (intramedullary rod) based on CPT fracture status and general health status. 6. 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. 7. Participant can provide an adequate ATC sample volume. 8. Participant weighs ≥5 kg/11 lb at Screening and on Day 1. 9. Participant is not pregnant or lactating. 10. If participant is of childbearing potential, is practicing highly effective methods of birth control from Screening to the end of the study: * Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation: * Oral * Intravaginal * Transdermal * Progestogen-only hormonal contraception associated with inhibition of ovulation: * Oral * Injectable * Implantable * Intrauterine device * Intrauterine hormone-releasing system * Bilateral tubal occlusion * Vasectomized partner * Sexual abstinence, defined as refraining from heterosexual intercourse during study participation, is acceptable if this is the participant's usual lifestyle; periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and the lactational amenorrhea method are not acceptable methods of contraception Note: A participant is considered to be of childbearing potential if they are postmenarchal and premenopausal, unless surgically sterile (permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy). 11. Participant and parent(s)/legal guardian(s) are able to understand all study information provided and are willing to return to the study facility for all visits, including follow-up evaluations.	1. Participant has bilateral CPT. 2. Participant has evidence of plexiform neurofibroma of any size or nodular fibroma ≥1.2 inches/3 cm on the ipsilateral leg. 3. Participant has a clinically significant infection at the fracture site or systemic infection. 4. Participant's CPT fracture involves the metaphysis (i.e., not limited to the diaphysis). 5. 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. 6. Participant has an autoimmune disease, with the exception of well-controlled type 1 diabetes or autoimmune thyroid disorders. 7. Participant has an active (malignant) tumor. 8. 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. 9. Participant has any chronic, ongoing, or planned use of medications that might affect bone metabolism or bone quality such as bisphosphonates, steroids, methotrexate, vitamin K antagonists, immunosuppressant therapy, or immunotherapy during the study. 10. Participant has any history of allergic reaction or any anticipated hypersensitivity to any anesthetic agent or any potential hypersensitivity to any of the components of the NVD003 graft (including the CMRL1066 formulation medium) or hypersensitivity related to other factors in the surgical process for the ICBG graft, such as anesthesia, medications, suture materials or fixation devices. 11. Participant has received any investigational product (including a device) within 60 days before enrollment in the study. 12. Participant would be concurrently enrolled in another clinical study while participating in this study. 13. Participant has any clinically significant hematologic, renal, hepatic, and coagulation laboratory abnormalities (i.e., complete blood count, prothrombin time/international normalized ratio, Chem-7, liver function tests, etc.). 14. Participant or participant's parent(s)/legal guardian(s) have an unstable condition (e.g., psychiatric disorder, a recent history of substance abuse) or is otherwise thought to be unreliable or incapable of complying with the requirements of the protocol.

Inclusion Criteria

Exclusion Criteria

1. 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.
2. Participant is of any sex, ≤17 years of age.
3. Participant has been diagnosed with CPT (with or without NF1).
4. Participant has a non-healing Paley type 3 or 4 diaphyseal fracture.4
5. Participant is a candidate for surgical treatment using an internal fixation approach (intramedullary rod) based on CPT fracture status and general health status.
6. 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.
7. Participant can provide an adequate ATC sample volume.
8. Participant weighs ≥5 kg/11 lb at Screening and on Day 1.
9. Participant is not pregnant or lactating.
10. If participant is of childbearing potential, is practicing highly effective methods of birth control from Screening to the end of the study:
* Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
* Oral
* Intravaginal
* Transdermal
* Progestogen-only hormonal contraception associated with inhibition of ovulation:
* Oral
* Injectable
* Implantable
* Intrauterine device
* Intrauterine hormone-releasing system
* Bilateral tubal occlusion
* Vasectomized partner
* Sexual abstinence, defined as refraining from heterosexual intercourse during study participation, is acceptable if this is the participant's usual lifestyle; periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and the lactational amenorrhea method are not acceptable methods of contraception Note: A participant is considered to be of childbearing potential if they are postmenarchal and premenopausal, unless surgically sterile (permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy).
11. Participant and parent(s)/legal guardian(s) are able to understand all study information provided and are willing to return to the study facility for all visits, including follow-up evaluations.

1. Participant has bilateral CPT.
2. Participant has evidence of plexiform neurofibroma of any size or nodular fibroma ≥1.2 inches/3 cm on the ipsilateral leg.
3. Participant has a clinically significant infection at the fracture site or systemic infection.
4. Participant's CPT fracture involves the metaphysis (i.e., not limited to the diaphysis).
5. 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.
6. Participant has an autoimmune disease, with the exception of well-controlled type 1 diabetes or autoimmune thyroid disorders.
7. Participant has an active (malignant) tumor.
8. 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.
9. Participant has any chronic, ongoing, or planned use of medications that might affect bone metabolism or bone quality such as bisphosphonates, steroids, methotrexate, vitamin K antagonists, immunosuppressant therapy, or immunotherapy during the study.
10. Participant has any history of allergic reaction or any anticipated hypersensitivity to any anesthetic agent or any potential hypersensitivity to any of the components of the NVD003 graft (including the CMRL1066 formulation medium) or hypersensitivity related to other factors in the surgical process for the ICBG graft, such as anesthesia, medications, suture materials or fixation devices.
11. Participant has received any investigational product (including a device) within 60 days before enrollment in the study.
12. Participant would be concurrently enrolled in another clinical study while participating in this study.
13. Participant has any clinically significant hematologic, renal, hepatic, and coagulation laboratory abnormalities (i.e., complete blood count, prothrombin time/international normalized ratio, Chem-7, liver function tests, etc.).
14. Participant or participant's parent(s)/legal guardian(s) have an unstable condition (e.g., psychiatric disorder, a recent history of substance abuse) or is otherwise thought to be unreliable or incapable of complying with the requirements of the protocol.

Contacts and Locations

Study Contact

Judy Ashworth, MD

CONTACT

224-713-6154

clinical@novadip.com

Lola TOME

CONTACT

+447468479913

regulatory@novadip.com

Study Locations (Sites)

LifeBridge Health - International Center for Limb Lengthening

Baltimore, Maryland, 21117

United States

Collaborators and Investigators

Sponsor: Novadip Biosciences

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-09-02

Study Completion Date2027-07

Study Record Updates

Study Start Date2025-09-02

Study Completion Date2027-07

Terms related to this study

Keywords Provided by Researchers

tissue engineered therapy
bone graft
autologous
pediatric
congenital
non-union
pseudarthrosis
cellular therapy

Additional Relevant MeSH Terms

Congenital Pseudarthrosis of Tibia