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

Brief Title

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

Official Title

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

Brief 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
    

Detailed 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.
    


Study Type

Observational


Primary Outcome

Pulmonary function and thoracic volume of TIS patients


Condition

Thoracic Insufficiency Syndrome (TIS)

Intervention

Synthes VEPTR (Vertical Expandable Prosthetic Titanium Rib)

Study Arms / Comparison Groups

 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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Device

Estimated Enrollment

7

Start Date

November 11, 2010

Completion Date

February 28, 2020

Primary Completion Date

March 25, 2018

Eligibility Criteria

        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.
      

Gender

All

Ages

6 Months - 17 Years

Accepts Healthy Volunteers

No

Contacts

Jeffrey Martus, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02241954

Organization ID

091371


Responsible Party

Principal Investigator

Study Sponsor

Vanderbilt University Medical Center


Study Sponsor

Jeffrey Martus, MD, Principal Investigator, Vanderbilt University Medical Center


Verification Date

May 2020