Idiopathic Scoliosis
Conditions
Keywords
Scoliosis, Idiopathic Scoliosis, Adolescent Idiopathic Scoliosis
Brief summary
This study will assess whether Anterior Vertebral Tethering is a safe and feasible method of anterior approach surgery for spinal deformity in pediatric idiopathic scoliosis.
Detailed description
Scoliosis is a condition in which the spine is deformed by a curvature in the coronal plane. It is generally associated with a twisting (axial plane) deformity as well. It can have a variety of underlying etiologies and the etiology is used to classify the types of scoliosis. Idiopathic scoliosis is sub-classified in two ways: by age of onset and by magnitude of deformity. Curves between 10 and 25 degrees are considered mild. Curves between 25 and 50 degrees are classified as moderate. Curves greater than 50 degrees are termed severe. The current standard of care for moderate scoliosis in patients with remaining growth is to utilize a thoracolumbosacral orthosis (TLSO brace) to prevent progression of deformity. The scientific evidence has supported the efficacy of this intervention in avoiding progression of the Cobb angle to 50 degrees or more. If treated with a TLSO brace, many idiopathic scoliosis patients would conceivably be subjected to years of brace wear and the cost and psychological factors inherent therein. Additional downsides of brace treatment include the potentially negative psychosocial impact of wearing an external sign of deformity during adolescence, a key period of emotional development. Prior research has identified negative psychosocial effects related to wearing a brace in children. Recent evidence has suggested that certain curve patterns will likely progress to 50 degrees or more, despite treatment with a TLSO brace. Sanders, et al. demonstrated a correlation of Cobb angle (greater than 35 degrees) and skeletal maturity (bone age 4 or less) to the risk of progression to 50 degrees or more, despite TLSO bracing. The evidence supports that the current practice of TLSO bracing is not an effective treatment to avoid progression to 50 degrees in these patients. It is on this population (thoracic Cobb angle greater than 35 degrees, bone age of 4 or less) that we intend to test the safety and feasibility of Anterior Vertebral Body Tethering to avoid curve progression to 50 degrees. The study intervention is surgical orthopedic implantation of the Anterior Vertebral Tether Device, by way of thoracoscopic surgery under general anesthesia. The primary outcome measures include assessments of safety of the insertion procedure and of the device, as well as the secondary measure of feasibility by determining the ability to successfully implant the investigational device.
Interventions
DEVICEAnterior Vertebral Tether
Vertebral body tethering through anterior thoracoscopic approach under general anesthesia and fluoroscopic guidance.
Sponsors
Patrick Cahill, MD
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
DEVICE_FEASIBILITY
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
8 Years to 16 Years
Healthy volunteers
No
Inclusion criteria
1. Males or females age 8 to 16 years old at time of enrollment (inclusive) 2. Diagnosis of idiopathic scoliosis 3. Sanders bone age of less than or equal to 4 4. Thoracic curve of greater than or equal to 35 degrees and less than or equal to 60 degrees 5. Lumbar curve less than 35 degrees 6. Patient has already been identified for and recommended to have surgical intervention 7. Spina bifida occulta is permitted 8. Spondylolysis or Spondylolisthesis is permitted, as long as it is non-operative, the subject has not had any previous surgery for this, and no surgery is planned in the future
Exclusion criteria
1. Pregnancy (current) 2. Prior spinal or chest surgery 3. MRI abnormalities (including syrinx greater than 4mm, Chiari malformation, or tethered cord) 4. Neuromuscular, thoracogenic, cardiogenic scoliosis, or any other non-idiopathic scoliosis 5. Associated syndrome, including Marfan syndrome or neurofibromatosis 6. Sanders bone age greater than 4 7. Thoracic curve less than 35 degrees or greater than 60 degrees 8. Lumbar curve greater than or equal to 35 degrees 9. Unable or unwilling to firmly commit to returning for required follow-up visits 10. Investigator judgement that the subject/family may not be a candidate for the intervention
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events | up to 2 years | Intra-operative and post-intervention medical events or signs and symptoms of complications arising after the start of study intervention will be captured. The event description, date of onset, end date, severity, and outcome will be documented. The frequencies, type, body system, severity, and relationship to the study intervention will also be summarized. A distinction will be made between those events which are device-related and non device-related. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Comparison of Pre-Operative and Post-Operative Cobb Angle | up to 2 years | The exploratory therapeutic endpoint used for feasibility will be change in post-operative Cobb angle compared to pre-operative Cobb angle, measured on coronal radiograph of the spine. |
| Comparison of Pre-Operative and Post-Operative SRS 30 Scores | up to 2 years | The Scoliosis Research Society 30 (SRS-30) questionnaire will be administered at pre-operative and post-operative time points. This questionnaire measures health-related quality of life outcomes. It consists of 30 questions divided into five domains: Function/Activity (5 questions), Pain (5 questions), Self-image/Appearance (5 questions), Mental Health (5 questions), and Satisfaction with management (2 questions). The remaining 8 questions are a combination of different domains. Each question is scored on a 5-point scale (1 = worst, 5 = best). To score each domain, sum the scores of the questions within that domain and then divide by the number of questions in that domain to get an average score. To calculate the total overall score, sum all the individual question scores and then divide by the total number of questions (30). Higher scores indicate better health-related quality of life. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Anterior Vertebral Tethering Subjects receiving Anterior Vertebral Tethering intervention.
Anterior Vertebral Tether: Vertebral body tethering through anterior thoracoscopic approach under general anesthesia and fluoroscopic guidance. | 20 |
| Total | 20 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Withdrawn due to need for re-operation related to an anticipated device SAE | 4 |
Baseline characteristics
| Characteristic | Anterior Vertebral Tethering |
|---|---|
| Age, Continuous | 12 years STANDARD_DEVIATION 1.94 |
| Age, Customized Age at first diagnosis of Idiopathic Scoliosis | 9 years STANDARD_DEVIATION 2.38 |
| Bending Lumbar Cobb Angle | 5 Degrees STANDARD_DEVIATION 9.83 |
| Bending Thoracic Cobb Angle | 20.5 Degrees STANDARD_DEVIATION 10.56 |
| Coronal Lumbar Cobb Angle | 29 Degrees STANDARD_DEVIATION 6.77 |
| Coronal Thoracic Cobb Angle | 51.5 Degrees STANDARD_DEVIATION 7.25 |
| Degree of lumbar rib hump | 6 Degrees STANDARD_DEVIATION 2.72 |
| Degree of thoracic rib hump | 13 Degrees STANDARD_DEVIATION 4.64 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 19 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Height (cm) | 159.4 cm STANDARD_DEVIATION 12.43 |
| Lumbar rib hump side Left | 20 Participants |
| Lumbar rib hump side Right | 0 Participants |
| Menarche status N/A, Male | 4 Participants |
| Menarche status No | 10 Participants |
| Menarche status Yes | 6 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 1 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 4 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 15 Participants |
| Region of Enrollment United States | 20 participants |
| Sanders Bone Age 0 | 0 Participants |
| Sanders Bone Age 1 | 0 Participants |
| Sanders Bone Age 2 | 2 Participants |
| Sanders Bone Age 3 | 10 Participants |
| Sanders Bone Age 4 | 8 Participants |
| Sanders Bone Age 5 | 0 Participants |
| Sanders Bone Age 6 | 0 Participants |
| Sanders Bone Age 7 | 0 Participants |
| Sanders Bone Age 8 | 0 Participants |
| Sex: Female, Male Female | 16 Participants |
| Sex: Female, Male Male | 4 Participants |
| Thoracic Rib Hump Side Left | 0 Participants |
| Thoracic Rib Hump Side Right | 20 Participants |
| Weight (kg) | 45.8 kg STANDARD_DEVIATION 13.82 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 20 |
| other Total, other adverse events | 20 / 20 |
| serious Total, serious adverse events | 6 / 20 |
Outcome results
Primary
Incidence of Treatment-Emergent Adverse Events
Intra-operative and post-intervention medical events or signs and symptoms of complications arising after the start of study intervention will be captured. The event description, date of onset, end date, severity, and outcome will be documented. The frequencies, type, body system, severity, and relationship to the study intervention will also be summarized. A distinction will be made between those events which are device-related and non device-related.
Time frame: up to 2 years
Population: Four of the subjects were withdrawn from the study prior to the 2 year completion date due to need for re-operation related to an anticipated device SAE. All data reported for these subjects were obtained prior to withdrawal.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Anterior Vertebral Tethering | Incidence of Treatment-Emergent Adverse Events | Subjects that experienced a device-related AE | 11 Participants |
| Anterior Vertebral Tethering | Incidence of Treatment-Emergent Adverse Events | Subjects that experienced a device-related SAE | 7 Participants |
| Anterior Vertebral Tethering | Incidence of Treatment-Emergent Adverse Events | Subjects that experienced a non-device related AE | 20 Participants |
| Anterior Vertebral Tethering | Incidence of Treatment-Emergent Adverse Events | Subjects that experienced a non-device related SAE | 0 Participants |
Secondary
Comparison of Pre-Operative and Post-Operative Cobb Angle
The exploratory therapeutic endpoint used for feasibility will be change in post-operative Cobb angle compared to pre-operative Cobb angle, measured on coronal radiograph of the spine.
Time frame: up to 2 years
Population: Four of the subjects were withdrawn from the study prior to the 2 year completion date due to need for re-operation related to an anticipated device SAE. All data reported for these subjects were obtained prior to withdrawal.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative Cobb Angle | Thoracic curve <50° without a secondary spinal fusion or other revision surgery | 14 Participants |
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative Cobb Angle | Thoracic curve >50°, or requiring a secondary spinal fusion or other revision surgery | 6 Participants |
Secondary
Comparison of Pre-Operative and Post-Operative SRS 30 Scores
The Scoliosis Research Society 30 (SRS-30) questionnaire will be administered at pre-operative and post-operative time points. This questionnaire measures health-related quality of life outcomes. It consists of 30 questions divided into five domains: Function/Activity (5 questions), Pain (5 questions), Self-image/Appearance (5 questions), Mental Health (5 questions), and Satisfaction with management (2 questions). The remaining 8 questions are a combination of different domains. Each question is scored on a 5-point scale (1 = worst, 5 = best). To score each domain, sum the scores of the questions within that domain and then divide by the number of questions in that domain to get an average score. To calculate the total overall score, sum all the individual question scores and then divide by the total number of questions (30). Higher scores indicate better health-related quality of life.
Time frame: up to 2 years
Population: Four of the 20 initial subjects were withdrawn from the study prior to final follow-up administration of SRS questionnaire.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative SRS 30 Scores | Pre-operative Function/Activity | 4.4 score on a scale | Standard Deviation 0.4 |
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative SRS 30 Scores | Pre-operative Pain | 4.3 score on a scale | Standard Deviation 0.7 |
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative SRS 30 Scores | Pre-operative Self-image/Appearance | 3.6 score on a scale | Standard Deviation 0.8 |
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative SRS 30 Scores | Pre-operative Mental Health | 4.2 score on a scale | Standard Deviation 0.6 |
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative SRS 30 Scores | Pre-operative Satisfaction with management | 3.5 score on a scale | Standard Deviation 0.9 |
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative SRS 30 Scores | Pre-operative total score | 4.1 score on a scale | Standard Deviation 0.5 |
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative SRS 30 Scores | Post-operative Function/Activity | 4.5 score on a scale | Standard Deviation 0.4 |
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative SRS 30 Scores | Post-operative Pain | 4.6 score on a scale | Standard Deviation 0.5 |
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative SRS 30 Scores | Post-operative Self-image/Appearance | 4.2 score on a scale | Standard Deviation 0.7 |
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative SRS 30 Scores | Post-operative Mental Health | 4.1 score on a scale | Standard Deviation 0.7 |
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative SRS 30 Scores | Post-operative Satisfaction with management | 4.2 score on a scale | Standard Deviation 1 |
| Anterior Vertebral Tethering | Comparison of Pre-Operative and Post-Operative SRS 30 Scores | Post-operative total score | 4.3 score on a scale | Standard Deviation 0.5 |