<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT02241954</url>
  </required_header>
  <id_info>
    <org_study_id>091371</org_study_id>
    <nct_id>NCT02241954</nct_id>
  </id_info>
  <brief_title>Vertical Expandable Prosthetic Titanium Rib (VEPTR) for Thoracic Insufficiency Syndrome</brief_title>
  <acronym>VEPTR</acronym>
  <official_title>Ertical Expandable Prosthetic Titanium Rib (VEPTR) for Thoracic Insufficiency Syndrome</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Vanderbilt University Medical Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Vanderbilt University Medical Center</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The hypothesis of the study is that individuals with poor pulmonary function secondary to TIS&#xD;
      will benefit from expansion thoracoplasty. The investigators specific aims are to:&#xD;
&#xD;
        1. Record pulmonary function and thoracic volume of TIS patients at enrollment and with&#xD;
           serial observation&#xD;
&#xD;
        2. Offer expansion thoracoplasty therapy to TIS patients with&#xD;
&#xD;
             1. poor pulmonary function at enrollment&#xD;
&#xD;
             2. progressive decline in pulmonary function&#xD;
&#xD;
             3. progressive chest wall and/or spinal deformity&#xD;
&#xD;
             4. lack of appropriate increase in thoracic volume during growth&#xD;
&#xD;
        3. Document efficacy of expansion thoracoplasty for TIS patients&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Study procedures&#xD;
&#xD;
        1. Patient Identification &amp; Enrollment Patients meeting the inclusion criteria will be&#xD;
           invited to enroll in the study as described above.&#xD;
&#xD;
        2. Serial clinical observation&#xD;
&#xD;
           Patients will be followed over time for progression of thoracic insufficiency&#xD;
           indicators:&#xD;
&#xD;
             -  Worsening respiratory status oFrequent respiratory infections oElevated resting&#xD;
                respiratory rate oEase of fatigability oHypoxia and hypercapnia oMarionette sign&#xD;
                (trunk and shoulder elevation and depression with respiration)&#xD;
&#xD;
             -  Primary diaphragmatic breathing (loss of chest wall mobility)&#xD;
&#xD;
             -  Progressive scoliosis&#xD;
&#xD;
             -  Failure of thoracic dimensions to increase with growth&#xD;
&#xD;
           Patients with progressive thoracic insufficiency will be offered surgical intervention&#xD;
           with expansion thoracoplasty utilizing the VEPTR device.&#xD;
&#xD;
           Studies which are standard of care for TIS will be utilized to assess patients. Routine&#xD;
           standard of care studies will be obtained:&#xD;
&#xD;
             1. MRI of the spine will be obtained at presentation.&#xD;
&#xD;
             2. Chest CT scan (every 2 years, or less frequently if clinically indicated)&#xD;
&#xD;
             3. AP/Lat spine xrays (to include the chest wall)&#xD;
&#xD;
             1. Will be obtained every 6 months - 12 months&#xD;
&#xD;
             2. If a patient has a VEPTR implantation, the typical schedule of radiographs would&#xD;
                include immediate postoperative xrays (the day of surgery and/or early&#xD;
                postoperative period) and at 6 weeks, 12 weeks, and 6 months postoperative.&#xD;
&#xD;
             3. VEPTR lengthening will typically take place every 6 months after the initial VEPTR&#xD;
                implantation.&#xD;
&#xD;
           i.Xrays will be obtained every 6 months in association with the VEPTR lengthening.&#xD;
&#xD;
           ii.Once VEPTR lengthening is no longer clinically indicated, the patients will be&#xD;
           followed with xrays every 6-24 months until skeletal maturity.&#xD;
&#xD;
           d.Pulmonary function tests (spirometry) will be obtained every 6-12 months in children&#xD;
           who are old enough to participate (typically &gt; age 4 years).&#xD;
&#xD;
           e.Arterial and/or capillary blood gases&#xD;
&#xD;
           a.Blood gases will be obtained every 6-12 months if clinically indicated and&#xD;
           perioperatively for VEPTR implantation / lengthening.&#xD;
&#xD;
           f.Routine vital signs including resting pulse oximetry (SaO2)&#xD;
&#xD;
           a.Will be assessed at clinic visits and perioperatively g.All of the above mentioned&#xD;
           studies will be obtained more or less frequently as clinically indicated.&#xD;
&#xD;
        3. Expansion thoracoplasty with VEPTR implantation and subsequent lengthening with the&#xD;
           VEPTR will be performed following informed consent. The surgical technique will be as&#xD;
           described by Campbell.3 The VEPTR device will be lengthened at 6 to 12 month intervals&#xD;
           until thoracic and spinal deformities have stabilized radiographically.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">November 11, 2010</start_date>
  <completion_date type="Actual">February 28, 2020</completion_date>
  <primary_completion_date type="Actual">March 25, 2018</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Pulmonary function and thoracic volume of TIS patients</measure>
    <time_frame>Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)</time_frame>
    <description>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.)</description>
  </primary_outcome>
  <primary_outcome>
    <measure>ffer expansion thoracoplasty therapy to TIS patients meeting certain criteria (provided in description below)</measure>
    <time_frame>Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)</time_frame>
    <description>Offer expansion thoracoplasty therapy to TIS patients with&#xD;
poor pulmonary function at enrollment&#xD;
progressive decline in pulmonary function&#xD;
progressive chest wall and/or spinal deformity&#xD;
lack of appropriate increase in thoracic volume during growth</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Document efficacy of expansion thoracoplasty for TIS patients</measure>
    <time_frame>Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)</time_frame>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">7</enrollment>
  <condition>Thoracic Insufficiency Syndrome (TIS)</condition>
  <arm_group>
    <arm_group_label>Expansion Thoracoplasty</arm_group_label>
    <description>The rib prosthesis is lengthened periodically to correct the concavity of the deformity, expanding the volume of the hypoplastic thorax and improving associated spinal deformity. This device has been FDA approved by a Humanitarian Device Exemption and is described as a Vertical Expandable Prosthetic Titanium Rib (VEPTR). An updated version of the VEPTR, the VEPTR II, may be used instead depending on the physician's preference and the patient's anatomy. The VEPTR II device has greater size range and modularity of implants than the original VEPTR.</description>
  </arm_group>
  <intervention>
    <intervention_type>Device</intervention_type>
    <intervention_name>Synthes VEPTR (Vertical Expandable Prosthetic Titanium Rib)</intervention_name>
    <arm_group_label>Expansion Thoracoplasty</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Pediatric patients, age range from 6 months to 17 years with thoracic insufficiency&#xD;
        syndrome (TIS). Categories of TIS include:&#xD;
&#xD;
          1. Flail chest syndrome&#xD;
&#xD;
          2. Constrictive chest wall syndrome (rib fusions and scoliosis)&#xD;
&#xD;
          3. Hypoplastic thorax syndrome (Jeune's syndrome, achondroplasia, Jarcho-levin syndrome,&#xD;
             Ellis van Creveld syndrome)&#xD;
&#xD;
          4. Progressive scoliosis/kyphosis of congenital, neurogenic, or idiopathic origin without&#xD;
             rib anomaly&#xD;
&#xD;
          5. Early onset scoliosis without rib anomaly at high risk for progression&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Pediatric patients, age range from 6 months - 17 years with thoracic insufficiency&#xD;
             syndrome (TIS) will be considered for inclusion. Categories of TIS include:&#xD;
&#xD;
               1. Flail chest syndrome&#xD;
&#xD;
               2. Constrictive chest wall syndrome (rib fusions and scoliosis)&#xD;
&#xD;
               3. Hypoplastic thorax syndrome (Jeune's syndrome, achondroplasia, Jarcho-levin&#xD;
                  syndrome, Ellis van Creveld syndrome)&#xD;
&#xD;
               4. Progressive scoliosis/kyphosis of congenital, neurogenic, or idiopathic origin&#xD;
                  without rib anomaly&#xD;
&#xD;
               5. Early onset scoliosis without rib anomaly at high risk for progression&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Failure to meet inclusion criteria.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Months</minimum_age>
    <maximum_age>17 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Jeffrey Martus, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Vanderbilt University Medical Center</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Monroe Carell Jr. Children's Hospital at Vanderbilt</name>
      <address>
        <city>Nashville</city>
        <state>Tennessee</state>
        <zip>37232</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2020</verification_date>
  <study_first_submitted>September 3, 2014</study_first_submitted>
  <study_first_submitted_qc>September 15, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 16, 2014</study_first_posted>
  <last_update_submitted>May 11, 2020</last_update_submitted>
  <last_update_submitted_qc>May 11, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">May 12, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Vanderbilt University Medical Center</investigator_affiliation>
    <investigator_full_name>Jeffrey Martus</investigator_full_name>
    <investigator_title>Assistant Professor of Orthopaedics</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Syndrome</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT02241954
org study id: 091371
nct id: NCT02241954
lead sponsor: 
has dmc: No
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
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.
observational model: Cohort
time perspective: Prospective
measure: Pulmonary function and thoracic volume of TIS patients
time frame: Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)
description: 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.)
measure: ffer expansion thoracoplasty therapy to TIS patients meeting certain criteria (provided in description below)
time frame: Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)
description: 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
measure: Document efficacy of expansion thoracoplasty for TIS patients
time frame: Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)
arm group label: Expansion Thoracoplasty
description: The rib prosthesis is lengthened periodically to correct the concavity of the deformity, expanding the volume of the hypoplastic thorax and improving associated spinal deformity. This device has been FDA approved by a Humanitarian Device Exemption and is described as a Vertical Expandable Prosthetic Titanium Rib (VEPTR). An updated version of the VEPTR, the VEPTR II, may be used instead depending on the physician's preference and the patient's anatomy. The VEPTR II device has greater size range and modularity of implants than the original VEPTR.
intervention type: Device
intervention name: Synthes VEPTR (Vertical Expandable Prosthetic Titanium Rib)
arm group label: Expansion Thoracoplasty
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 6 Months
maximum age: 17 Years
healthy volunteers: No
last name: Jeffrey Martus, MD
role: Principal Investigator
affiliation: Vanderbilt University Medical Center
facility: 
country: United States
responsible party type: Principal Investigator
investigator affiliation: Vanderbilt University Medical Center
investigator full name: Jeffrey Martus
investigator title: Assistant Professor of Orthopaedics
mesh term: Syndrome
sharing ipd: No