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

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

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.

Study Overview

• Status: Completed
• Conditions: Neuromuscular Scoliosis; Distraction System; Growth Friendly System
• Intervention / Treatment: Device: SDS; Device: NEMOST

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 Type: Interventional
• Enrollment (Actual): 28
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Netherlands
  • Rotterdam, Netherlands
    • Erasmus Medical Center
  • Utrecht, Netherlands, 3584CX
    • UMC Utrecht
  • Noord-Holland
    • Amsterdam, Noord-Holland, Netherlands, 1105AZ
      • Amsterdam UMC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Spring Distraction System (SDS); The SDS will be placed and fits around a standard rod of 5.5mm.	Device: SDS; The patient is implanted with SDS.
Experimental: Necker Enfants Malade OSTeosynthesis (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	Device: NEMOST; The patient is implanted with NEMOST.

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: UMC Utrecht
• Collaborators: EUROS
• Investigators: Principal Investigator: Moyo C Kruyt, MD, PhD, UMC Utrecht

Publications and helpful links

Helpful Links

• Surgical treatment of neuromuscular Early Onset Scoliosis with a bilateral posterior one-way rod compared to the Spring Distraction System: study protocol for a limited-efficacy Randomized Controlled Trial (BiPOWR)

Study record dates

Study Major Dates

• Study Start (Actual): May 6, 2019
• Primary Completion (Actual): March 13, 2023
• Study Completion (Actual): March 13, 2023

Study Registration Dates

• First Submitted: June 13, 2019
• First Submitted That Met QC Criteria: July 15, 2019
• First Posted (Actual): July 16, 2019

Study Record Updates

• Last Update Posted (Actual): June 5, 2023
• Last Update Submitted That Met QC Criteria: June 1, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Spinal Diseases; Bone Diseases; Spinal Curvatures; Scoliosis
• Other Study ID Numbers: NL 64018.041.17

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No