Limited-efficacy Testing of Spring Distraction System (SDS) and Unilateral One-way Rod (MID-C) for Early Onset Scoliosis (UniPOWR)

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.

Study Overview

• Status: Withdrawn
• Conditions: Idiopathic Scoliosis; Early-Onset Scoliosis Deformity of Spine (Disorder)
• Intervention / Treatment: Device: SDS; Device: MID-C

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.

• Study Type: Interventional
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Netherlands
  • Utrecht, Netherlands, 3584 CX
    • UMC Utrecht
  • Noord-Holland
    • Amsterdam, Noord-Holland, Netherlands, 1105 AZ
      • Amsterdam UMC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 12 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

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; The SDS device will be implanted during a scoliosis correction operation.	Device: SDS; The SDS will be added to a traditional growing rod (TGR) of 4.5 or 5.5mm.
Experimental: Minimal Invasive Deformity Correction system; The MID-C device will be implanted during a scoliosis correction operation.	Device: MID-C; The MID-C system consists of a unilateral one-way ratchet rod that is connected to the spine with polyaxial connectors

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Limited-efficacy of SDS and MID-C in terms of curve correction maintenance: changes in cobb angle	changes in cobb angle on radiographs post-op and at 4 weeks, 3 months and 12 months follow-up (FU). A maximum of 5 degrees increase will be the threshold to define maintenance.	Until 1 year post-operative FU
Incidence of Treatment-Emergent Serious Adverse Events of SDS and MID-C	Reported treatment related Serious Adverse Events (SAEs) per-operatively and at 4 weeks, 3 months and 12 months FU.	Until 1 year post-operative FU

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Limited-efficacy of SDS and MID-C 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 and 12 months FU.	Until 1 year post-operative FU
SDS vs. MID-C 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 and 12 months FU. A maximum of 5 degrees increase will be the threshold to define maintenance.	Until 1 year post-operative FU
SDS vs. MID-C 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 and 12 months FU.	Until 1 year post-operative FU
SDS vs. MID-C with respect to the incidence of Treatment-Emergent Serious Adverse Events	Reported treatment related SAEs per-operatively and at 4 weeks, 3 months and 12 months FU.	Until 1 year post-operative FU
SDS vs. MID-C with respect to surgery time	Surgery time in minutes	Until 1 year post-operative FU
SDS vs. MID-C with respect to blood loss during surgery	blood loss in cc	Until 1 year post-operative FU
SDS vs. MID-C with respect to length of hospital stay	length of hospital stay in days	Until 1 year post-operative FU
SDS vs. MID-C with respect to recovery time	recovery time in minutes	Until 1 year post-operative FU
SDS vs. MID-C with respect to bone density	Bone density changes of the bypassed vertebrae on Dual Energy X-ray Absorptiometry (DEXA)scan post-op and at 3 months en 12 months FU.	Until 1 year post-operative FU
SDS vs. MID-C 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 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-operative FU
SDS vs. MID-C with respect to flexibility of the spine	Flexibility and 3D rotation of the spine on dynamic echography (Scolioscan)	Until 1 year post-operative FU
SDS vs. MID-C with respect to 3D development of the spine	Apical Vertebral Rotation based on MRI	Until 1 year post-operative FU
SDS vs. MID-C with respect to patient appearance	sagittal balance on clinical photographs post-op and at 1 year FU	Until 1 year post-operative FU

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Anticipated): July 1, 2019
• Primary Completion (Anticipated): July 1, 2022
• Study Completion (Anticipated): December 1, 2022

Study Registration Dates

• First Submitted: June 7, 2019
• First Submitted That Met QC Criteria: June 28, 2019
• First Posted (Actual): July 1, 2019

Study Record Updates

• Last Update Posted (Actual): March 23, 2020
• Last Update Submitted That Met QC Criteria: March 18, 2020
• Last Verified: March 1, 2020

More Information

Terms related to this study

• Keywords: Growth friendly system; Distraction system; Early Onset Scoliosis; Unilateral
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Spinal Diseases; Bone Diseases; Spinal Curvatures; Scoliosis
• Other Study ID Numbers: NL63511.041.17

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