Clinical trial NCT04021784: Limited-efficacy Testing of SDS and NEMOST for Early Onset Neuromuscular Scoliosis
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
Overall status: COMPLETED
Phase: NA
Conditions: Neuromuscular Scoliosis
Distraction System
Growth Friendly System
Lead sponsor: UMC Utrecht
Sponsor class: OTHER
Collaborators: name: EUROS
First posted: 2019-07-16
Enrollment: 28
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Half of the patients will be implanted with SDS system, the other half will be implanted with NEMOST.
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Limited-efficacy of SDS and NEMOST in terms of curve correction maintenance
timeFrame: Until 1 year post-operatively
measure: Incidence of possible Treatment-Emergent Serious Adverse Events of SDS and NEMOST
timeFrame: Until 1 year post-operatively
Secondary outcomes:
measure: Limited-efficacy of SDS and NEMOST in terms of spinal length
timeFrame: Until 1 year post-operatively
measure: SDS vs. NEMOST with respect to limited-efficacy in terms of curve correction maintenance
timeFrame: Until 1 year post-operatively
measure: SDS vs. NEMOST with respect to limited-efficacy in terms of spinal length
timeFrame: Until 1 year post-operatively
measure: SDS vs. NEMOST with respect to the development of the sagittale profile and instrumented thoracic kyphosis
timeFrame: Until 1 year post-operatively
measure: SDS vs. NEMOST with respect to surgery time
timeFrame: Until 1 year post-operatively
measure: SDS vs. NEMOST with respect to blood loss during surgery
timeFrame: Until 1 year post-operatively
measure: SDS vs. NEMOST with respect to length of hospital stay
timeFrame: Until 1 year post-operatively
measure: SDS vs. NEMOST with respect to recovery time
timeFrame: Until 1 year post-operatively
measure: SDS vs. NEMOST with respect to the incidence of disease- or treatment-related Serious Adverse Events
timeFrame: Until 1 year post-operatively
measure: SDS vs. NEMOST with respect to 3D development of the spine
timeFrame: Pre-operatively
measure: SDS vs. NEMOST with respect to Quality Of Life (QOL) on the Early Onset Scoliosis Questionnaires (EOSQ-24)
timeFrame: Until 1 year post-operatively
measure: SDS vs. NEMOST with respect to the effect on the development of the pelvic obliquity
timeFrame: Until 1 year post-operatively
Other outcomes:
Locations:
city: Amsterdam
country: Netherlands
facility: Amsterdam UMC
state: Noord-Holland
city: Rotterdam
country: Netherlands
facility: Erasmus Medical Center
city: Utrecht
country: Netherlands
facility: UMC Utrecht
Study officials:
affiliation: UMC Utrecht
name: Moyo C Kruyt, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: