Clinical trial NCT04003233: Limited-efficacy Testing of Spring Distraction System (SDS) and Unilateral One-way Rod (MID-C) for Early Onset Scoliosis
Official title: Limited-efficacy Testing of Spring Distraction System (SDS) and Unilateral One-way Rod (MID-C) for Early Onset Scoliosis
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: NA
Conditions: Early-Onset Scoliosis Deformity of Spine (Disorder)
Idiopathic Scoliosis
Keywords: Growth friendly system
Distraction system
Early Onset Scoliosis
Unilateral
Lead sponsor: UMC Utrecht
Sponsor class: OTHER
Collaborators: name: Apifix
First posted: 2019-07-01
Enrollment: 0
Sex: ALL
Minimum age (years): 6
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Participants will be randomized and assigned to either SDS or MID-C.
Primary purpose: TREATMENT

Brief summary: The primary aim of this study is to investigate the limited efficacy of these innovative surgical solutions in treatment of Early Onset Scoliosis (EOS) in terms of maintaining reduction while maintaining spinal growth. The secondary aim is to compare both devices for these and other parameters as well as safety.

Detailed description: Rationale: EOS is a severe and potentially life-threatening disorder since a disturbance of spinal development leads to impaired development of the trunk, with cardio-respiratory failure as a result, often early in adult life. Several innovative solutions have been developed to treat growing children with severe EOS. The SDS device was developed internally at the dpt. of orthopedics at the UMC Utrecht, the Netherlands, the MID-C device was developed by the company ApiFix Ltd.

Objective: The primary aim of this study is to investigate the limited efficacy of these innovative surgical solutions in treatment of early onset scoliosis in terms of maintaining reduction while maintaining spinal growth. The secondary aim is to compare both devices for these and other parameters as well as safety.

Study design: A feasibility study using two prospective cohorts according to an open label randomized clinical trial (RCT) design. The study will be done in two tertiary referral centers (UMC Utrecht and Amsterdam UMC). Primary endpoints are maintenance of curve correction as well as complications. These data will be compared to a recently described cohort of patients that received a "standard treatment" (Magnetically controlled growing rod (MCGR)). The same endpoints as well as secondary outcomes, will be compared between the two new treatments.

Study population: Children with idiopathic(-like) early onset scoliosis with an indication for a growing rod implant.

Eligibility criteria: Inclusion Criteria:

* Ambulant skeletally immature children, 6-12 yrs. of age, with open triradiate cartilages on X-ray
* Scoliosis diagnosis prior to the age 10
* Diagnosis of idiopathic or mild syndromic scoliosis (e.g. 22q11DS, Trisomy 21 or 9, Coffin-Siris)
* Progressive scoliosis qualified for growth system surgery
* One curve for treatment with an apex below Th5 and a proximal end vertebra below Th2
* The primary curve must be between 35 and 75 degrees coronal Cobb angle
* The primary curve must be non-rigid (i.e. the curve reduces on bending X-rays to \<35 degrees or reduces \>30% )
* Normal or hypokyphotic sagittal alignment (Th5 -Th12 \< 50 degrees) on lateral X-rays

Exclusion Criteria:

* Patients with an obvious neuromuscular disease
* Patients that are severely mentally retarded
* Patients with a scoliosis that extends to the pelvis or the cervicothoracic region
* Patients with a main curve of more than 8 vertebra Cobb to Cobb
* Patients with a skeletal dysplasia that effects growth (e.g. achondroplasia, SED)
* Patients with a systemic disease which severely influences bone quality (e.g. osteogenesis imperfecta, metabolic diseases)
* Patients with soft tissue weakness (e.g. Ehler Danlos, Marfan, Neurofibromatosis, Prader Willi)
* Patients with an active systemic disease such as Juvenile Idiopathic Arthritis, HIV or oncologic treatment
* Patients with a previous surgical fusion of the spine

Primary outcomes:
measure: Limited-efficacy of SDS and MID-C in terms of curve correction maintenance: changes in cobb angle
timeFrame: Until 1 year post-operative FU
measure: Incidence of Treatment-Emergent Serious Adverse Events of SDS and MID-C
timeFrame: Until 1 year post-operative FU
Secondary outcomes:
measure: Limited-efficacy of SDS and MID-C in terms of spinal length
timeFrame: Until 1 year post-operative FU
measure: SDS vs. MID-C with respect to limited-efficacy in terms of curve correction maintenance
timeFrame: Until 1 year post-operative FU
measure: SDS vs. MID-C with respect to limited-efficacy in terms of spinal length
timeFrame: Until 1 year post-operative FU
measure: SDS vs. MID-C with respect to the incidence of Treatment-Emergent Serious Adverse Events
timeFrame: Until 1 year post-operative FU
measure: SDS vs. MID-C with respect to surgery time
timeFrame: Until 1 year post-operative FU
measure: SDS vs. MID-C with respect to blood loss during surgery
timeFrame: Until 1 year post-operative FU
measure: SDS vs. MID-C with respect to length of hospital stay
timeFrame: Until 1 year post-operative FU
measure: SDS vs. MID-C with respect to recovery time
timeFrame: Until 1 year post-operative FU
measure: SDS vs. MID-C with respect to bone density
timeFrame: Until 1 year post-operative FU
measure: SDS vs. MID-C with respect to Quality Of Life (QOL) on the Early Onset Scoliosis Questionnaires (EOSQ-24)
timeFrame: Until 1 year post-operative FU
measure: SDS vs. MID-C with respect to flexibility of the spine
timeFrame: Until 1 year post-operative FU
measure: SDS vs. MID-C with respect to 3D development of the spine
timeFrame: Until 1 year post-operative FU
measure: SDS vs. MID-C with respect to patient appearance
timeFrame: Until 1 year post-operative FU
Other outcomes:
Locations:
city: Amsterdam
country: Netherlands
facility: Amsterdam UMC
state: Noord-Holland
city: Utrecht
country: Netherlands
facility: UMC Utrecht
Study officials:
affiliation: UMC Utrecht
name: Moyo C Kruyt, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: