Non-interventional Study in Pediatric Patients Treated With NOVOCART Inject in the Knee

Study Overview
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Brief Summary
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The study is a prospective, multicenter, single-arm non-interventional study to evaluate the efficacy, safety and health economics of NOVOCART® Inject in the treatment of cartilage defects of the knee in pediatric patients with radiologically proven closed epiphyseal growth plates.

Detailed Description
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In this non-interventional study NOVOCART® Inject, which is marketed in Germany under a license according to Section 4b of the AMG, will be used in routine clinical practice according to the current authorized Summary of Product Characteristics (SmPC). As stated in this SmPC, NOVOCART® Inject may only be used by physicians who are trained on the product. The treatment with NOVOCART® Inject requires two surgeries. During the first surgery cartilage tissue will be harvested arthroscopically and sent to TETEC AG for NOVOCART® Inject manufacturing, then NOVOCART® Inject will be transplanted during a second surgery about 3 to 4 weeks later. All patients will be followed up for 5 years post NOVOCART® Inject implantation and data will be collected preoperatively and at 3, 6, 12, 18, 24, 36, 48 and 60 months as outlined at the end of this synopsis. NOVOCART® Inject post treatment rehabilitation should follow the recommendations given in the SmPC and during product training. The primary and secondary variables (see below) will be assessed after a follow-up of 24 month post NOVOCART® Inject implantation (primary analysis). Long-term data will be assessed after an additional 3 years follow-up (follow-up analysis). The collected data will be documented using dedicated case report forms (CRFs) which are created by TETEC AG.

Official Title
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Prospective, Non-interventional Study to Evaluate the Efficacy and Safety of NOVOCART Inject for the Treatment of Cartilage Defects in the Knee in Pediatric Patients With Closed Epiphyses

Conditions
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Cartilage Disease

Intervention / Treatment
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* Drug: NOVOCART Inject


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Pediatric patients (< 18 years of age at implantation) Closure of the epiphysis adjacent to the biopsy harvest site or the defect to be treated confirmed by an adequate imaging modality Localized full-thickness cartilage defect (ICRS grade III or IV) of the knee Medicinal indication for NOVOCART® Inject treatment Exclusion Criteria: see NOVOCART Inject SmPC

Ages Eligible for Study
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Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: NOVOCART Inject|Autologous Chondrocyte Implantation|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Responder rate of overall KOOS | The overall Knee injury and Osteoarthritis Outcome Score (KOOS5) responder rate defined as the proportion of patients with ≥ 10 points improvement in the KOOS5 from baseline. | 24-months Follow-up | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change of overall KOOS from baseline | Change of overall KOOS from baseline to the 24-month assessment, Knee Injury and Osteoarthritis Outcome Score, score 0-100, higher score shows better outcome | 24-months Follow-up | 
| Change of the 5 sub-scores of the KOOS from baseline | Change of the 5 sub-scores of the KOOS from baseline to the 24-month assessment, Knee Injury and Osteoarthritis Outcome Score, score 0-100, higher score shows better outcome | 24-months Follow-up | 
| Change of the IKDC subjective score from baseline | Change from baseline to the 24-month visit in the IKDC subjective score, international knee documentation committee, score 0-100, higher score shows better outcome | 24-months Follow-up | 
| Change in the IKDC surgeon's part from baseline | Change from baseline to the 24-month visit in the IKDC surgeon's part. international knee documentation committee, score 0-100, higher score shows better outcome | 24-months Follow-up | 
| MOCART (MRI imaging) | In vivo performance measured by the assessment of the Magnetic Resonance, Observation of Cartilage Repair Tissue (MOCART) score, Magnetic Resonance Observation of Cartilage Repair Tissue, score 0-100, higher score shows better outcome | 24-months Follow-up | 
| IKDC subjective score responder rate | IKDC subjective score responder rate, defined as the proportion of patients with > 20.5 points improvement in the IKDC subjective score from baseline to the 24-month visit | 24-months Follow-up | 
| Proportion of patients with treatment failure | Proportion of patients with treatment failure | 24-months Follow up, 60-months Follow-up | 
| Number of postop physical therapy | Number of postop physical therapy | 24-months Follow up, 60-months Follow-up | 
| Number of postop doctor visits | Number of postop doctor visits | 24-months Follow up, 60-months Follow-up | 
| Days of hospitalisation | Days of hospitalisation | 24-months Follow up, 60-months Follow-up | 
| Work/school status checklist | Work/school status, changes due to injury in work or school status (pupil, worker full or part time, not working, job seeking) | 24-months Follow up, 60-months Follow-up | 
| Days of work/school missed | Days of work/school missed | 24-months Follow up, 60-months Follow-up | 
| Patient satisfaction | Patient questionnaire, 4 questions with 3 answer choices better, same, worse | 24-months Follow up, 60-months Follow-up | 
| Treatment-related adverse reactions (ARs) | Treatment-related adverse reactions (ARs) | 24-months Follow up, 60-months Follow-up | 
| Rate of unplanned re-operations | Rate of unplanned re-operations: all unplanned reoperations and those related to NOVOCART® Inject treatment | 24-months Follow up, 60-months Follow-up | 
| Surgical time | cut-to-suture time | 24-months Follow up, 60-months Follow-up | 
| Length of incision | Length of incision | 24-months Follow up, 60-months Follow-up | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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NOVOCART Inject, Cartilage Defect, Pediatric Patients, Knee, Cartilage Injury