Vertical Expandable Prosthetic Titanium Rib (VEPTR) for Thoracic Insufficiency Syndrome

Thoracic Insufficiency Syndrome (TIS) Clinical Trial

— VEPTR  

Official title:

Ertical Expandable Prosthetic Titanium Rib (VEPTR) for Thoracic Insufficiency Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02241954
• Other study ID #: 091371
• Status: Completed
• Start date: November 11, 2010
• Est. completion date: February 28, 2020

Study information

• Verified date: May 2020
• Source: Vanderbilt University Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The hypothesis of the study is that individuals with poor pulmonary function secondary to TIS will benefit from expansion thoracoplasty. The investigators specific aims are to:

1. Record pulmonary function and thoracic volume of TIS patients at enrollment and with serial observation

2. Offer expansion thoracoplasty therapy to TIS patients with

1. poor pulmonary function at enrollment

2. progressive decline in pulmonary function

3. progressive chest wall and/or spinal deformity

4. lack of appropriate increase in thoracic volume during growth

3. Document efficacy of expansion thoracoplasty for TIS patients

Description:

Study procedures

1. Patient Identification & Enrollment Patients meeting the inclusion criteria will be invited to enroll in the study as described above.

2. Serial clinical observation

Patients will be followed over time for progression of thoracic insufficiency indicators:

• Worsening respiratory status oFrequent respiratory infections oElevated resting respiratory rate oEase of fatigability oHypoxia and hypercapnia oMarionette sign (trunk and shoulder elevation and depression with respiration)

• Primary diaphragmatic breathing (loss of chest wall mobility)

• Progressive scoliosis

• Failure of thoracic dimensions to increase with growth

Patients with progressive thoracic insufficiency will be offered surgical intervention with expansion thoracoplasty utilizing the VEPTR device.

Studies which are standard of care for TIS will be utilized to assess patients. Routine standard of care studies will be obtained:

1. MRI of the spine will be obtained at presentation.

2. Chest CT scan (every 2 years, or less frequently if clinically indicated)

3. AP/Lat spine xrays (to include the chest wall)

1. Will be obtained every 6 months - 12 months

2. If a patient has a VEPTR implantation, the typical schedule of radiographs would include immediate postoperative xrays (the day of surgery and/or early postoperative period) and at 6 weeks, 12 weeks, and 6 months postoperative.

3. VEPTR lengthening will typically take place every 6 months after the initial VEPTR implantation.

i.Xrays will be obtained every 6 months in association with the VEPTR lengthening.

ii.Once VEPTR lengthening is no longer clinically indicated, the patients will be followed with xrays every 6-24 months until skeletal maturity.

d.Pulmonary function tests (spirometry) will be obtained every 6-12 months in children who are old enough to participate (typically > age 4 years).

e.Arterial and/or capillary blood gases

a.Blood gases will be obtained every 6-12 months if clinically indicated and perioperatively for VEPTR implantation / lengthening.

f.Routine vital signs including resting pulse oximetry (SaO2)

a.Will be assessed at clinic visits and perioperatively g.All of the above mentioned studies will be obtained more or less frequently as clinically indicated.

3. Expansion thoracoplasty with VEPTR implantation and subsequent lengthening with the VEPTR will be performed following informed consent. The surgical technique will be as described by Campbell.3 The VEPTR device will be lengthened at 6 to 12 month intervals until thoracic and spinal deformities have stabilized radiographically.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 7
• Est. completion date: February 28, 2020
• Est. primary completion date: March 25, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 17 Years

Eligibility

Inclusion Criteria:

• Pediatric patients, age range from 6 months - 17 years with thoracic insufficiency syndrome (TIS) will be considered for inclusion. Categories of TIS include:

1. Flail chest syndrome

2. Constrictive chest wall syndrome (rib fusions and scoliosis)

3. Hypoplastic thorax syndrome (Jeune's syndrome, achondroplasia, Jarcho-levin syndrome, Ellis van Creveld syndrome)

4. Progressive scoliosis/kyphosis of congenital, neurogenic, or idiopathic origin without rib anomaly

5. Early onset scoliosis without rib anomaly at high risk for progression

Exclusion Criteria:

• Failure to meet inclusion criteria.

Related Conditions & MeSH terms

• Syndrome
• Thoracic Insufficiency Syndrome (TIS)

Intervention

Device:
• Synthes VEPTR (Vertical Expandable Prosthetic Titanium Rib)

Locations

Country	Name	City	State
United States	Monroe Carell Jr. Children's Hospital at Vanderbilt	Nashville	Tennessee

Sponsors (1)

Lead Sponsor	Collaborator
Vanderbilt University Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Pulmonary function and thoracic volume of TIS patients	1)Record pulmonary function and thoracic volume of TIS patients at enrollment and with serial observation postoperatively (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)	Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)
Primary	ffer expansion thoracoplasty therapy to TIS patients meeting certain criteria (provided in description below)	Offer expansion thoracoplasty therapy to TIS patients with; poor pulmonary function at enrollment; progressive decline in pulmonary function; progressive chest wall and/or spinal deformity; lack of appropriate increase in thoracic volume during growth	Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)
Primary	Document efficacy of expansion thoracoplasty for TIS patients		Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)