Autologous adipose-derived stem cells for Congenital pseudarthrosis of the tibia|Congenital pseudarthrosis

Inclusion criteria

• {"criterion_text":"- 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.\n- 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: o Oral o Intravaginal o Transdermal • Progestogen-only hormonal contraception associated with inhibition of ovulation: o Oral o Injectable o 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). If participant is sexually active and has a partner who may become pregnant (i.e., neither surgically sterile nor postmenopausal), agrees to use highly effective contraception (e.g., sterilization, birth control pills, Depo Provera injections, or contraceptive implants) from Screening to the end of the study. Participant agrees to refrain from donating sperm or eggs from Screening to the end of the study.\n- 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.\n- Participant is of any sex, ≤17 years of age.\n- Participant has been diagnosed with CPT (with or without NF1).\n- Participant has a non-healing Paley type 3 or 4 diaphyseal fracture.\n- Participant is a candidate for surgical treatment using an internal fixation approach (intramedullary rod) based on CPT fracture status and general health status.\n- 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.\n- Participant can provide an adequate ATC sample volume.\n- Participant weighs ≥5 kg/11 lb at Screening and on Day 1.\n- Participant is not pregnant or lactating."}

Exclusion criteria

• {"criterion_text":"- Participant has bilateral CPT.\n- 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.\n- Participant has received any investigational product (including a device) within 60 days before enrollment in the study.\n- Participant would be concurrently enrolled in another clinical study while participating in this study.\n- 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.).\n- 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.\n- Participant has evidence of plexiform neurofibroma of any size or nodular fibroma ≥1.2 inches/3 cm on the ipsilateral leg.\n- Participant has a clinically significant infection at the fracture site or systemic infection.\n- Participant’s CPT fracture involves the metaphysis (i.e., not limited to the diaphysis).\n- 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.\n- Participant has an autoimmune disease, with the exception of well-controlled type 1 diabetes or autoimmune thyroid disorders.\n- Participant has an active (malignant) tumor.\n- 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.\n- 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. Note that perioperative treatment with a bisphosphonate is allowed in Cohort A participants randomized to ICBG if its use is deemed SOC by the treating surgeon."}

Primary endpoints

• {"endpoint_text":"- 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)","definition_or_measurement_approach":"Binary (yes/no) overall healing at 12 months derived from 3 components; radiographic healing defined as mRUST score ≥13 assessed by independent central readers (ICRs)."}
• {"endpoint_text":"- Clinical healing measured by the absence of pain with weight-bearing, based on clinician’s assessment.","definition_or_measurement_approach":"Clinician assessment of absence of pain with weight-bearing at 12 months."}
• {"endpoint_text":"- 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.","definition_or_measurement_approach":"Assessment of whether any secondary interventions to promote/accelerate bone healing were used; combined with the other two components to determine binary overall healing."}

Secondary endpoints

• {"endpoint_text":"- 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.","definition_or_measurement_approach":"Radiographic and clinical healing and use of secondary interventions assessed at 12 months post-surgery (radiographic centrally-read; clinical by clinician)."}
• {"endpoint_text":"- • 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.","definition_or_measurement_approach":"Safety endpoints include counts and severity of AEs/SAEs related to NVD003; perioperative blood transfusion units; duration of grafting surgery (hours); hospital length of stay (days); recurrent fracture within 12 months."}
• {"endpoint_text":"- • 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.","definition_or_measurement_approach":"Radiological union assessed centrally using eLSS at 3, 6, 12 months; functional walking by timed 10-meter walk when appropriate at same timepoints."}
• {"endpoint_text":"- • 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.","definition_or_measurement_approach":"QoL measured by PODCI parent-report for children ≥2 years; mean change from baseline at 6 and 12 months."}

Spain

Earliest CTIS Part Ii Submission Date

28-07-2025

Latest Decision Or Authorization Date

10-09-2025

Processing Time Days

44

Number Of Sites

1

Number Of Participants

1

Belgium

Earliest CTIS Part Ii Submission Date

28-07-2025

Latest Decision Or Authorization Date

07-11-2025

Processing Time Days

102

Number Of Sites

1

Number Of Participants

2

France

Earliest CTIS Part Ii Submission Date

28-07-2025

Latest Decision Or Authorization Date

10-03-2026

Processing Time Days

225

Number Of Sites

2

Number Of Participants

1

Primary sponsor

Full Name

Novadip Biosciences

Organisation Type

Pharmaceutical company

Country Of Registered Address

Belgium

Contract research organisations

Name

Premier Research Group S.L.

Responsibilities

Sponsor third party listed with contact email PremierCommunications@premier-research.com and sponsorDuties code 5

Third parties

• {"country":"Spain","full_name":"Premier Research Group S.L.","duties_or_roles":"5","organisation_type":"Pharmaceutical company"}