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出境医 / 临床实验 / Limited-efficacy Testing of SDS and NEMOST for Early Onset Neuromuscular Scoliosis (BiPOWR)
Limited-efficacy Testing of SDS and NEMOST for Early Onset Neuromuscular Scoliosis (BiPOWR)
Study Description
Brief Summary:
The primary aim of this study is to investigate 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 investigate growth of the instrumented spine and to compare these devices.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Neuromuscular Scoliosis Distraction System Growth Friendly System | Device: SDS Device: NEMOST | Not Applicable |
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 done in two centers (UMC Utrecht and Amsterdam UMC).
The 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 patient performance based on the EOSQ 24 and Lenke questionnaire, surgical parameters, maintenance of curve mobility by using ultrasound and pulmonary function
Study Design
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 28 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Intervention Model Description: | Half of the patients get the SDS system and the other half the NEMOST. |
| Masking: | Single (Participant) |
| Masking Description: | The patient is blinded to the medical device till after surgery. This due to the fact that we make x-rays and then the patients gets to see which medical device is implanted. |
| Primary Purpose: | Treatment |
| Official Title: | Limited-efficacy Testing of Spring Distraction System (SDS) and a Bilateral One Way Rod (NEMOST) for Early Onset Neuromuscular Scoliosis (BiPOWR) |
| Actual Study Start Date : | May 6, 2019 |
| Estimated Primary Completion Date : | May 2022 |
| Estimated Study Completion Date : | December 2022 |
Arms and Interventions
| Arm | Intervention/treatment |
|---|---|
|
Experimental: Spring Distraction System (SDS)
The SDS will be placed and fits around a standard rod of 4.5 or 5.5mm.
|
Device: SDS
The patient gets a SDS implanted.
|
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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 gets a NEMOST implanted.
|
Outcome Measures
Primary Outcome Measures :
- Limited-efficacy of SDS and NEMOST in terms of curve correction maintenance [ Time Frame: Until 1 year post-operative FU ]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 [ Time Frame: Until 1 year post-operative ]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 [ Time Frame: Until 1 year post-operative FU ]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 [ Time Frame: Until 1 year post-operative FU ]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 [ Time Frame: Until 1 year post-operative FU ]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 [ Time Frame: Until 1 year post-operative FU ]Changes in sagittale 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 [ Time Frame: Until 1 year post-operative FU ]Surgery time in minutes
- SDS vs. NEMOST with respect to blood loss during surgery [ Time Frame: Until 1 year post-operative FU ]Blood loss in cc
- SDS vs. NEMOST with respect to length of hospital stay [ Time Frame: Until 1 year post-operative FU ]Length of hospital stay in days
- SDS vs. NEMOST with respect to recovery time [ Time Frame: Until 1 year post-operative FU ]Recovery time in minutes
- SDS vs. NEMOST with respect to the incidence of Treatment-Emergent Serious Adverse Events [ Time Frame: Until 1 year post-operative FU ]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 [ Time Frame: Pre-operatively ]Apical Vertebral Rotation based on bone MRI
- SDS vs. NEMOST with respect to flexibility of the spine [ Time Frame: Until 1 year post-operative FU ]Flexibility and 3D rotation of the spine on dynamic echography (Scolioscan)
- SDS vs. NEMOST with respect to patient appearance [ Time Frame: Until 1 year post-operative FU ]Sagittal balance on clinical photographs post-op and at 1 year FU
- SDS vs. NEMOST with respect to Quality Of Life (QOL) on the Early Onset Scoliosis Questionnaires (EOSQ-24) [ Time Frame: Until 1 year post-operative FU ]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 de lungfunction of the patient [ Time Frame: Until 1 year post-operative FU ]The lungfunction is tested by spirometry (Forced vital capacity (FVC)) (UMC Utrecht only).
- SDS vs. NEMOST with respect to the effect on the development of the pelvic incidence (PI) [ Time Frame: Until 1 year post-operative FU ]Changes in pelvic incidence on X-rays
- SDS vs. NEMOST with respect to the attitude of patients in wheelchair [ Time Frame: Until 1 year post-operative FU ]Changes in attitude of the patients in the wheelchair based on clinical performance.
Eligibility Criteria
| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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 (at least 25% decrease on bending X rays)
- 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.
Contacts and Locations
Contacts
| Contact: Moyo C Kruyt, MD, PhD | +31887551133 | m.c.kruyt@umcutrecht.nl | |
| Contact: Justin Lemans | +31887556445 | J.V.C.Lemans-3@umcutrecht.nl |
Locations
| Netherlands | |
| Amsterdam UMC | Recruiting |
| Amsterdam, Noord-Holland, Netherlands, 1105AZ | |
| Contact: Agnita Stadhouder, MD +31 20 56 62672 a.stadhouder@amsterdamumc.nl | |
| UMC Utrecht | Recruiting |
| Utrecht, Netherlands, 3584CX | |
| Contact: Moyo Kruyt, MD, PhD +31887551133 m.c.kruyt@umcutrecht.nl | |
| Contact: Justin Lemans +31887556445 J.V.C.Lemans-3@umcutrecht.nl | |
Sponsors and Collaborators
UMC Utrecht
EUROS
Investigators
| Principal Investigator: | Moyo C Kruyt, MD, PhD | UMC Utrecht |
| Tracking Information | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| First Submitted Date ICMJE | June 13, 2019 | ||||||||
| First Posted Date ICMJE | July 16, 2019 | ||||||||
| Last Update Posted Date | May 4, 2021 | ||||||||
| Actual Study Start Date ICMJE | May 6, 2019 | ||||||||
| Estimated Primary Completion Date | May 2022 (Final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures ICMJE |
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| Original Primary Outcome Measures ICMJE | Same as current | ||||||||
| Change History | |||||||||
| Current Secondary Outcome Measures ICMJE |
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| Original Secondary Outcome Measures ICMJE |
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| Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title ICMJE | Limited-efficacy Testing of SDS and NEMOST for Early Onset Neuromuscular Scoliosis | ||||||||
| Official Title ICMJE | Limited-efficacy Testing of Spring Distraction System (SDS) and a Bilateral One Way Rod (NEMOST) for Early Onset Neuromuscular Scoliosis (BiPOWR) | ||||||||
| Brief Summary | The primary aim of this study is to investigate 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 investigate growth of the instrumented spine and to compare these 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 done in two centers (UMC Utrecht and Amsterdam UMC). The 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 patient performance based on the EOSQ 24 and Lenke questionnaire, surgical parameters, maintenance of curve mobility by using ultrasound and pulmonary function |
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| Study Type ICMJE | Interventional | ||||||||
| Study Phase ICMJE | Not Applicable | ||||||||
| Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Intervention Model Description: Half of the patients get the SDS system and the other half the NEMOST. Masking: Single (Participant)Masking Description: The patient is blinded to the medical device till after surgery. This due to the fact that we make x-rays and then the patients gets to see which medical device is implanted. Primary Purpose: Treatment
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| Condition ICMJE |
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| Intervention ICMJE |
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| Study Arms ICMJE |
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| Publications * | Not Provided | ||||||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status ICMJE | Recruiting | ||||||||
| Estimated Enrollment ICMJE |
28 | ||||||||
| Original Estimated Enrollment ICMJE | Same as current | ||||||||
| Estimated Study Completion Date ICMJE | December 2022 | ||||||||
| Estimated Primary Completion Date | May 2022 (Final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria ICMJE |
Inclusion Criteria:
Exclusion Criteria:
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| Sex/Gender ICMJE |
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| Ages ICMJE | Child, Adult, Older Adult | ||||||||
| Accepts Healthy Volunteers ICMJE | No | ||||||||
| Contacts ICMJE |
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| Listed Location Countries ICMJE | Netherlands | ||||||||
| Removed Location Countries | |||||||||
| Administrative Information | |||||||||
| NCT Number ICMJE | NCT04021784 | ||||||||
| Other Study ID Numbers ICMJE | NL 64018.041.17 | ||||||||
| Has Data Monitoring Committee | Yes | ||||||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement ICMJE |
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| Responsible Party | M.C. Kruyt, MD, PhD, UMC Utrecht | ||||||||
| Study Sponsor ICMJE | UMC Utrecht | ||||||||
| Collaborators ICMJE | EUROS | ||||||||
| Investigators ICMJE |
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| PRS Account | UMC Utrecht | ||||||||
| Verification Date | May 2021 | ||||||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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