Limited-efficacy Testing of SDS and NEMOST for Early Onset Neuromuscular Scoliosis

Study Overview
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Brief Summary
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The primary aim of this study is to investigate and describe the limited efficacy of the Spring Distraction System (SDS) and Bilateral One Way Rod (NEMOST) in maintaining curve reduction without repeat lengthening procedures and complications. Secondary aims are to describe growth of the instrumented spine, health-related quality of life, and to compare both devices.

Detailed Description
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Several innovative solutions have been developed to treat growing children with a severe scoliosis. One device (SDS) was developed internally at the department of orthopaedics UMC Utrecht in the Netherlands. The other device (NEMOST) was developed at the Necker Hospital in France. This study is designed as a multicenter, limited-efficacy study using two prospective cohorts according a randomised clinical trial. The study will be performed in two centers (UMC Utrecht, Amsterdam UMC and Erasmus MC). Primary endpoints are the limited-efficacy in terms of maintenance of curve correction and occurrence of SAEs related to the procedure. These data will be compared to a recently described cohort of patients that received a standard treatment, the MGCR (Skov et al., 2017). Secondary endpoints include spinal- and implant growth, patient performance based on the EOSQ 24 questionnaire and surgical parameters.

Official Title
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Limited-efficacy Testing of Spring Distraction System (SDS) and a Bilateral One Way Rod (NEMOST) for Early Onset Neuromuscular Scoliosis (BiPOWR)

Conditions
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Neuromuscular Scoliosis, Distraction System, Growth Friendly System

Intervention / Treatment
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* Device: SDS
* Device: NEMOST


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Non ambulant Neuromuscular or syndromal scoliosis Progressive scoliosis indicated for bipolar fixation extending to the pelvis Diagnosis of scoliosis before age 10 Patient under 12 and open triradiate cartilage (usually closes around 12 years for girls and 14 years for boys) Main curve proximal end vertebra below Th 3 Non rigid curve Patients who have an indication for a primary surgery Exclusion Criteria: Ambulant Patients with closed triradiate cartilage Patients with a skeletal dysplasia that effects growth (Achondroplasia, SED) Patients with a systemic disease which severely influences bone quality e.g. osteogenesis imperfecta, metabolic diseases Patients with soft tissue weakness (Ehler Danlos, Marfan, Neurofibromatosis, Prader Willi) Patients that have a congenital anomaly of the spine of more than 5 vertebrae Patients with an active systemic disease such as JIA, HIV, oncologic treatment Patients with a previous surgical fusion of the spine Patients that are expected to be lost to FU due to e.g. likely to immigrate within 1 year. Patients that have had a previous spine surgery.
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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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Half of the patients will be implanted with SDS system, the other half will be implanted with NEMOST.
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Spring Distraction System (SDS)<br>The SDS will be placed and fits around a standard rod of 5.5mm. | Device: SDS<br>* The patient is implanted with SDS.<br>|
| Experimental: Necker Enfants Malade OSTeosynthesis (NEMOST)<br>The NEMOST is a one-way-rod that uses a ratchet type of locking mechanism. Both NEMOST devices should be placed in parallel, on the two fixator rods that are connected with a cross connector | Device: NEMOST<br>* The patient is implanted with NEMOST.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Limited-efficacy of SDS and NEMOST in terms of curve correction maintenance | Changes in cobb angle on radiographs post-op and at 4 weeks, 3 months, 6 months and 12 months follow-up (FU). A maximum of 5 degrees increase will be the threshold to define maintenance. | Until 1 year post-operatively | 
| Incidence of possible Treatment-Emergent Serious Adverse Events of SDS and NEMOST | Reported treatment related Serious Adverse Events (SAEs) per-operatively and at 4 weeks, 3 months, 6 months and 12 months FU. | Until 1 year post-operatively | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Limited-efficacy of SDS and NEMOST in terms of spinal length | Changes in length of T1-T12, T1-S1 and the instrumented segment in mm on calibrated Anterior Posterior (AP) X-rays post-op and at 4 weeks, 3 months, 6 months and 12 months FU. | Until 1 year post-operatively | 
| SDS vs. NEMOST with respect to limited-efficacy in terms of curve correction maintenance | Changes in cobb angle on radiographs post-op and at 4 weeks, 3 months, 6 months and 12 months follow-up (FU). A maximum of 5 degrees increase will be the threshold to define maintenance. | Until 1 year post-operatively | 
| SDS vs. NEMOST with respect to limited-efficacy in terms of spinal length | Changes in length of T1-T12, T1-S1 and the instrumented segment in mm on calibrated AP X-rays post-op and at 4 weeks, 3 months, 6 months and 12 months FU. | Until 1 year post-operatively | 
| SDS vs. NEMOST with respect to the development of the sagittale profile and instrumented thoracic kyphosis | Changes in sagittal profile and thoracic kyphosis and the instrumented (T1 pelvic angle, TPA) segment in mm on calibrated Anterior Posterior (AP) X-rays post-op and at 4 weeks, 3 months, 6 months and 12 months FU. | Until 1 year post-operatively | 
| SDS vs. NEMOST with respect to surgery time | Surgery time in minutes | Until 1 year post-operatively | 
| SDS vs. NEMOST with respect to blood loss during surgery | Blood loss in cc | Until 1 year post-operatively | 
| SDS vs. NEMOST with respect to length of hospital stay | Length of hospital stay in days | Until 1 year post-operatively | 
| SDS vs. NEMOST with respect to recovery time | Recovery time in minutes | Until 1 year post-operatively | 
| SDS vs. NEMOST with respect to the incidence of disease- or treatment-related Serious Adverse Events | Reported treatment related SAEs per-operatively and at 4 weeks, 3 months, 6 months and 12 months FU. | Until 1 year post-operatively | 
| SDS vs. NEMOST with respect to 3D development of the spine | Apical Vertebral Rotation based on bone MRI | Pre-operatively | 
| SDS vs. NEMOST with respect to Quality Of Life (QOL) on the Early Onset Scoliosis Questionnaires (EOSQ-24) | Parent reported QOL and performance is assessed with the 24-item Early Onset Scoliosis Questionnaires (EOSQ-24) pre-op and at 4 weeks, 3 months, 6 months and 12 months FU. EOSQ-24 covers the following domains: Child's Health Related Quality of Life (16 items), Family Impact (2 items) and Satisfaction (2 items). | Until 1 year post-operatively | 
| SDS vs. NEMOST with respect to the effect on the development of the pelvic obliquity | Changes in pelvic obliquity on X-rays | Until 1 year post-operatively |