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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novadip Biosciences

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16], Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

7 Nov 2025 → ongoing

Decision date (initial)

2025-09-08

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Novadip Biosciences

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To assess the efficacy of NVD003 for the surgical treatment of CPT in pediatric participants.

Secondary objectives 4

1. Efficacy: To assess the efficacy of NVD003 with respect to individual components of overall healing.
2. Safety: To assess the safety of NVD003 for the surgical treatment of CPT in pediatric participants.
3. To further evaluate the efficacy of NVD003 for the surgical treatment of CPT in pediatric participants based on other measures of bone healing.
4. To evaluate the effects of NVD003 on quality of life (QoL).

Conditions and MedDRA coding

Congenital pseudarthrosis of the tibia

Study design 1 period

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-000026-PIP80-61

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 11

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.
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: 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.
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.

Exclusion criteria 14

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. 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.
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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. 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)
2. Clinical healing measured by the absence of pain with weight-bearing, based on clinician’s assessment.
3. 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.

Secondary endpoints 4

1. 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.
2. • 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.
3. • 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.
4. • 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.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novadip Biosciences

Sponsor organisation

Novadip Biosciences

Address

Rue Granbonpre 11

City

Mont-Saint-Guibert

Postcode

1435

Country

Belgium

Scientific contact point

Organisation

Novadip Biosciences

Contact name

Chief Executive Officer

Public contact point

Organisation

Novadip Biosciences

Contact name

Chief Executive Officer

Third parties 1

Locations

3 EU/EEA countries · 4 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 53 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications