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

Not Applicable

Completed

• Conditions: Distraction System; Growth Friendly System; Neuromuscular Scoliosis
• Interventions: Device: NEMOST; Device: SDS

• Registration Number: NCT04021784
• Lead Sponsor: UMC Utrecht

Brief Summary

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

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.

Study Timeline

• Study Start: 2019-05-06
• Study First Submitted: 2019-06-13
• Study First Submitted QC: 2019-07-15
• Study First Posted: 2019-07-16
• Primary Completion: 2023-03-13
• Study Completion: 2023-03-13
• Status Verified: 2023-06
• Last Update Submitted: 2023-06-01
• Last Update Posted: 2023-06-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 28

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Necker Enfants Malade OSTeosynthesis (NEMOST)	NEMOST	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
Spring Distraction System (SDS)	SDS	The SDS will be placed and fits around a standard rod of 5.5mm.

Primary Outcome Measures

Name	Time	Method
Limited-efficacy of SDS and NEMOST in terms of curve correction maintenance	Until 1 year post-operatively	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.
Incidence of possible Treatment-Emergent Serious Adverse Events of SDS and NEMOST	Until 1 year post-operatively	Reported treatment related Serious Adverse Events (SAEs) per-operatively and at 4 weeks, 3 months, 6 months and 12 months FU.

Secondary Outcome Measures

Name	Time	Method
SDS vs. NEMOST with respect to blood loss during surgery	Until 1 year post-operatively	Blood loss in cc
Limited-efficacy of SDS and NEMOST in terms of spinal length	Until 1 year post-operatively	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.
SDS vs. NEMOST with respect to limited-efficacy in terms of curve correction maintenance	Until 1 year post-operatively	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.
SDS vs. NEMOST with respect to limited-efficacy in terms of spinal length	Until 1 year post-operatively	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.
SDS vs. NEMOST with respect to the development of the sagittale profile and instrumented thoracic kyphosis	Until 1 year post-operatively	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.
SDS vs. NEMOST with respect to surgery time	Until 1 year post-operatively	Surgery time in minutes
SDS vs. NEMOST with respect to length of hospital stay	Until 1 year post-operatively	Length of hospital stay in days
SDS vs. NEMOST with respect to the incidence of disease- or treatment-related Serious Adverse Events	Until 1 year post-operatively	Reported treatment related SAEs per-operatively and at 4 weeks, 3 months, 6 months and 12 months FU.
SDS vs. NEMOST with respect to Quality Of Life (QOL) on the Early Onset Scoliosis Questionnaires (EOSQ-24)	Until 1 year post-operatively	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).
SDS vs. NEMOST with respect to the effect on the development of the pelvic obliquity	Until 1 year post-operatively	Changes in pelvic obliquity on X-rays
SDS vs. NEMOST with respect to recovery time	Until 1 year post-operatively	Recovery time in minutes
SDS vs. NEMOST with respect to 3D development of the spine	Pre-operatively	Apical Vertebral Rotation based on bone MRI

Trial Locations

Locations (3)

Amsterdam UMC; 🇳🇱 Amsterdam, Noord-Holland, Netherlands

UMC Utrecht; 🇳🇱 Utrecht, Netherlands

Erasmus Medical Center; 🇳🇱 Rotterdam, Netherlands