Limited-efficacy Testing of SDS and NEMOST for Early Onset Neuromuscular Scoliosis (BiPOWR)
Primary Purpose
Neuromuscular Scoliosis, Distraction System, Growth Friendly System
Status
Completed
Phase
Not Applicable
Locations
Netherlands
Study Type
Interventional
Intervention
SDS
NEMOST
Sponsored by
About this trial
This is an interventional treatment trial for Neuromuscular Scoliosis
Eligibility Criteria
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.
Sites / Locations
- Amsterdam UMC
- Erasmus Medical Center
- UMC Utrecht
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Spring Distraction System (SDS)
Necker Enfants Malade OSTeosynthesis (NEMOST)
Arm Description
The SDS will be placed and fits around a standard rod of 5.5mm.
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
Outcomes
Primary Outcome Measures
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.
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.
Secondary Outcome Measures
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.
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.
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.
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.
SDS vs. NEMOST with respect to surgery time
Surgery time in minutes
SDS vs. NEMOST with respect to blood loss during surgery
Blood loss in cc
SDS vs. NEMOST with respect to length of hospital stay
Length of hospital stay in days
SDS vs. NEMOST with respect to recovery time
Recovery time in minutes
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.
SDS vs. NEMOST with respect to 3D development of the spine
Apical Vertebral Rotation based on bone MRI
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).
SDS vs. NEMOST with respect to the effect on the development of the pelvic obliquity
Changes in pelvic obliquity on X-rays
Full Information
NCT ID
NCT04021784
First Posted
June 13, 2019
Last Updated
June 1, 2023
Sponsor
UMC Utrecht
Collaborators
EUROS
1. Study Identification
Unique Protocol Identification Number
NCT04021784
Brief Title
Limited-efficacy Testing of SDS and NEMOST for Early Onset Neuromuscular Scoliosis
Acronym
BiPOWR
Official Title
Limited-efficacy Testing of Spring Distraction System (SDS) and a Bilateral One Way Rod (NEMOST) for Early Onset Neuromuscular Scoliosis (BiPOWR)
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
May 6, 2019 (Actual)
Primary Completion Date
March 13, 2023 (Actual)
Study Completion Date
March 13, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
UMC Utrecht
Collaborators
EUROS
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neuromuscular Scoliosis, Distraction System, Growth Friendly System
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Model Description
Half of the patients will be implanted with SDS system, the other half will be implanted with NEMOST.
Masking
Participant
Masking Description
The patient is blinded to the medical device until after surgery. This is done to prevent selection bias through withdrawal. Patients are unblinded post-operatively, to enable accurate patient counseling with radiographs, on which the implant can be clearly seen.
Allocation
Randomized
Enrollment
28 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Spring Distraction System (SDS)
Arm Type
Experimental
Arm Description
The SDS will be placed and fits around a standard rod of 5.5mm.
Arm Title
Necker Enfants Malade OSTeosynthesis (NEMOST)
Arm Type
Experimental
Arm Description
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
Intervention Type
Device
Intervention Name(s)
SDS
Intervention Description
The patient is implanted with SDS.
Intervention Type
Device
Intervention Name(s)
NEMOST
Intervention Description
The patient is implanted with NEMOST.
Primary Outcome Measure Information:
Title
Limited-efficacy of SDS and NEMOST in terms of curve correction maintenance
Description
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.
Time Frame
Until 1 year post-operatively
Title
Incidence of possible Treatment-Emergent Serious Adverse Events of SDS and NEMOST
Description
Reported treatment related Serious Adverse Events (SAEs) per-operatively and at 4 weeks, 3 months, 6 months and 12 months FU.
Time Frame
Until 1 year post-operatively
Secondary Outcome Measure Information:
Title
Limited-efficacy of SDS and NEMOST in terms of spinal length
Description
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.
Time Frame
Until 1 year post-operatively
Title
SDS vs. NEMOST with respect to limited-efficacy in terms of curve correction maintenance
Description
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.
Time Frame
Until 1 year post-operatively
Title
SDS vs. NEMOST with respect to limited-efficacy in terms of spinal length
Description
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.
Time Frame
Until 1 year post-operatively
Title
SDS vs. NEMOST with respect to the development of the sagittale profile and instrumented thoracic kyphosis
Description
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.
Time Frame
Until 1 year post-operatively
Title
SDS vs. NEMOST with respect to surgery time
Description
Surgery time in minutes
Time Frame
Until 1 year post-operatively
Title
SDS vs. NEMOST with respect to blood loss during surgery
Description
Blood loss in cc
Time Frame
Until 1 year post-operatively
Title
SDS vs. NEMOST with respect to length of hospital stay
Description
Length of hospital stay in days
Time Frame
Until 1 year post-operatively
Title
SDS vs. NEMOST with respect to recovery time
Description
Recovery time in minutes
Time Frame
Until 1 year post-operatively
Title
SDS vs. NEMOST with respect to the incidence of disease- or treatment-related Serious Adverse Events
Description
Reported treatment related SAEs per-operatively and at 4 weeks, 3 months, 6 months and 12 months FU.
Time Frame
Until 1 year post-operatively
Title
SDS vs. NEMOST with respect to 3D development of the spine
Description
Apical Vertebral Rotation based on bone MRI
Time Frame
Pre-operatively
Title
SDS vs. NEMOST with respect to Quality Of Life (QOL) on the Early Onset Scoliosis Questionnaires (EOSQ-24)
Description
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).
Time Frame
Until 1 year post-operatively
Title
SDS vs. NEMOST with respect to the effect on the development of the pelvic obliquity
Description
Changes in pelvic obliquity on X-rays
Time Frame
Until 1 year post-operatively
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Moyo C Kruyt, MD, PhD
Organizational Affiliation
UMC Utrecht
Official's Role
Principal Investigator
Facility Information:
Facility Name
Amsterdam UMC
City
Amsterdam
State/Province
Noord-Holland
ZIP/Postal Code
1105AZ
Country
Netherlands
Facility Name
Erasmus Medical Center
City
Rotterdam
Country
Netherlands
Facility Name
UMC Utrecht
City
Utrecht
ZIP/Postal Code
3584CX
Country
Netherlands
12. IPD Sharing Statement
Plan to Share IPD
No
Links:
URL
https://link.springer.com/article/10.1186/s12891-022-06048-4
Description
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)
Learn more about this trial
Limited-efficacy Testing of SDS and NEMOST for Early Onset Neuromuscular Scoliosis
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