Osteogenesis Imperfecta
Conditions
Keywords
nailing, nail, Osteogenesis, pediatric, bone, rare disease, brittle, telescopic
Brief summary
This study has been planned as part of the Orthofix Srl post-market active surveillance plan for the collection of data on both the clinical performance and the safety profile of the JTIN. The rationale of the proposed study is to update and support the pre-market clinical evaluation of the JTIN with Real World Evidence clinical data in a real-life surgical setting, in order to confirm the benefit/risk ratio of this medical device and to keep the CE mark under Medical Device Regulation (MDR) requirements. One selected site, experienced in the treatment of pediatric patients with OI, where the usage of JTIN is already part of the normal clinical practice, will participate in this study.
Detailed description
One selected site, experienced in the treatment of pediatric patients with OI, where the usage of JTIN is already part of the normal clinical practice, will participate in this study. Investigator will screen patients treated (or planned to be treated) with JTIN to verify inclusion and exclusion criteria, to achieve the enrollment of 25 cases. With lost to follow-up percentage estimated at 20%, this will lead to a total of 20 evaluable cases, i.e. 20 implanted JTIN (some patients may contribute for more than one implant). Patients are prospectively and retrospectively enrolled in the study: they may have undergone JTIN implantation from the 1st of January 2022. Enrolled patients, since this study is non-interventional and observational, will follow the standard medical practice of the site: no requirements regarding the treatment of patients will be imposed on the site or Principal Investigator and the Sponsor will not in any manner influence the treatment decisions. Data of enrolled subjects will be collected for this study up to 1 year from surgery. The hospital standard care usually, but not exclusively, includes: surgery, discharge and plaster removal visits, and then 3 other follow up visits up to 1 year from surgery (see "Visits and Assessments Schedule"). Visits frequency is estimated as average of the site normal clinical practice, actual visit timing for each patient will be performed according to investigators and hospital staff evaluation. The patient data will be systematically collected by the investigator in eCRF.
Interventions
DEVICEJTIN implantation surgery
The JTIN is a self-extending intramedullary nail (telescopic rod) designed to provide fixation of femoral and/or tibial fractures, osteotomies, malunions and non-unions. The design of the JTIN includes a female and a male component, which are anchored to the proximal cortex and distal epiphysis through screw-type fixation. These two components slide from each other, allowing for extension of the JTIN as the bone structures remodel and patient growth occurs.
Sponsors
Orthofix s.r.l.
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Months to 18 Years
Healthy volunteers
No
Inclusion criteria
* is in pediatric age (\> 18 month and \< 18 years) at the time of surgery; * is skeletally immature; * has a diagnosis for OI; * has a regular indication for surgical intervention with JTIN to treat femoral and/or tibial fractures, osteotomies, malunions and non-unions; * patient and/or legal representative is duly informed and doesn't oppose to participation.
Exclusion criteria
* has a medical condition that is a contraindication according to the manufacturer's instruction for use; * has any conditions that in the Investigator's opinion may interfere with the study execution or due to which the patient should not participate for safety reasons; * requires the application of, or has already in-situ the application of concomitant devices that cannot be safely removed (except for permitted concomitant devices paragraph); * is participating in other clinical trials or has taken part in any clinical study in the last 3 months with exception of analytical trials on genetics study related to OI (i.e. studies that do not include an investigational treatment for the patient such as new drugs or other medical devices); * is likely to be lost to follow up, according to investigator's opinion.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Procedures With at Least One Serious/Not Serious Adverse Event Certainly or Possibly Related to JTIN | 1 year | The primary endpoint is measured to evaluate the clinical safety profile of JTIN within the scope of its intended purpose. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Implant Survival Rate: Percentage of Not Exchanged Nails | 1 year | This endpoint measures how many nails, in percentage, remains functional in the patient without requiring surgical replacement. Specifically, it represents the proportion of implanted nails that do not need to be exchanged during the follow-up period for any reason. A higher implant survival rate indicates better durability, safety for patients and clinical performance. This endpoint is measured to evaluate the clinical safety of JTIN. |
| Percentage of Procedures With Bone Union Achievement | 1 year | This endpoint is measured to evaluate the clinical performance of JTIN. |
| Post-treatment Fracture-free Survival Rate | 1 year | This endpoint is measured to evaluate the clinical performance of JTIN. This endpoint is described with the percentage of patients that experienced a fracture of the JTIN-treated bone during the specified time frame. |
Countries
France
Contacts
PRINCIPAL_INVESTIGATORZagorka Pejin, MD
Necker-Enfants Malades Hospital
Participant flow
Recruitment details
The patient information and the collection of the non-opposition form could also be carried out after the patient's treatment, as the study is entirely observational and allows for the inclusion of retrospective patients. This was the case for all patients included in the study: all had undergone surgery with JTIN before the study began and were therefore informed about the study after their treatment.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous Age at study inclusion | 7.5 years STANDARD_DEVIATION 3 |
| Age, Continuous Age at surgery | 5.4 years STANDARD_DEVIATION 3 |
| Region of Enrollment France | 14 Participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 9 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 14 |
| other Total, other adverse events | 10 / 14 |
| serious Total, serious adverse events | 9 / 14 |
Outcome results
None listed