Thoracic Insufficiency Syndrome (TIS)VEPTRNCT02241954
Vertical Expandable Prosthetic Titanium Rib (VEPTR) for Thoracic Insufficiency Syndrome
An observational study of Thoracic Insufficiency Syndrome (TIS), run by Vanderbilt University Medical Center at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 6 months to 17 years
- Conditions the study is for
- Thoracic Insufficiency Syndrome (TIS)
- What is being tested?
- Synthes VEPTR (Vertical Expandable Prosthetic Titanium Rib)
- Type of treatment
- Medical device
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Nov 2010to 28 Feb 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Pediatric patients, age range from 6 months to 17 years with thoracic insufficiency syndrome (TIS). 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
Who may be able to join
- Pediatric patients, age range from 6 months - 17 years with thoracic insufficiency syndrome (TIS) will be considered for inclusion. Categories of TIS include:
- Flail chest syndrome
- Constrictive chest wall syndrome (rib fusions and scoliosis)
- Hypoplastic thorax syndrome (Jeune's syndrome, achondroplasia, Jarcho-levin syndrome, Ellis van Creveld syndrome)
- Progressive scoliosis/kyphosis of congenital, neurogenic, or idiopathic origin without rib anomaly
- Early onset scoliosis without rib anomaly at high risk for progression
Who cannot join
- Failure to meet inclusion criteria.
Trial locations and contacts
1 site in 1 country.
United States1 site
Monroe Carell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232
About this study
Official title: Ertical Expandable Prosthetic Titanium Rib (VEPTR) for Thoracic Insufficiency Syndrome
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:
- Record pulmonary function and thoracic volume of TIS patients at enrollment and with serial observation
- 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
- Document efficacy of expansion thoracoplasty for TIS patients
Read the detailed description
Study procedures
- Patient Identification \& Enrollment Patients meeting the inclusion criteria will be invited to enroll in the study as described above.
- 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:
- MRI of the spine will be obtained at presentation.
- Chest CT scan (every 2 years, or less frequently if clinically indicated)
- AP/Lat spine xrays (to include the chest wall) <!-- -->
- Will be obtained every 6 months - 12 months
- 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.
- 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.
- 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.
- Conditions
- Thoracic Insufficiency Syndrome (TIS)
- Being tested
- Synthes VEPTR (Vertical Expandable Prosthetic Titanium Rib)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Synthes VEPTR (Vertical Expandable Prosthetic Titanium Rib)Device
| Study group | Role | What they receive |
|---|---|---|
| Expansion Thoracoplasty | 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. Receives: Device: Synthes VEPTR (Vertical Expandable Prosthetic Titanium Rib) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.)
Measured: Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)
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
Measured: Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)
Document efficacy of expansion thoracoplasty for TIS patients
Measured: Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2014 (estimated)
- Study start11 Nov 2010
- Primary completion25 Mar 2018
- Study completion28 Feb 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thoracic Insufficiency Syndrome (TIS)Studies of the same condition, open and under way.
- More trials run by Vanderbilt University Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jeffrey Martus, Vanderbilt University Medical Center
- Investigators
- Jeffrey Martus, MD, Principal investigator, Vanderbilt University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02241954
- Sponsor's protocol ID
- 091371
- Acronym
- VEPTR
- Status last verified
- May 2020
- First submitted
- 3 Sep 2014
- Last updated
- 12 May 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ertical Expandable Prosthetic Titanium Rib (VEPTR) for Thoracic Insufficiency Syndrome
- ClinicalTrials.gov ID
- NCT02241954
- Acronym / sponsor ID
- VEPTR / 091371
- Conditions
- Thoracic Insufficiency Syndrome (TIS)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Vanderbilt University Medical CenterUniversity, hospital or other
- Study start
- 11 Nov 2010 (actual)
- Primary completion
- 25 Mar 2018 (actual)
- Study completion
- 28 Feb 2020 (actual)
- First posted
- 16 Sep 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- Record pulmonary function and thoracic volume of TIS patients at enrollment and with serial observation
- 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
- Document efficacy of expansion thoracoplasty for TIS patients
Detailed description
Study procedures
- Patient Identification \& Enrollment Patients meeting the inclusion criteria will be invited to enroll in the study as described above.
- 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:
- MRI of the spine will be obtained at presentation.
- Chest CT scan (every 2 years, or less frequently if clinically indicated)
- AP/Lat spine xrays (to include the chest wall) <!-- -->
- Will be obtained every 6 months - 12 months
- 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.
- 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.
- 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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Expansion Thoracoplasty | – | Device: Synthes VEPTR (Vertical Expandable Prosthetic Titanium Rib)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. |
Who may qualify
- Ages
- 6 months to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Pediatric patients, age range from 6 months to 17 years with thoracic insufficiency syndrome (TIS). 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
Key inclusion criteria (6 listed)
- Pediatric patients, age range from 6 months - 17 years with thoracic insufficiency syndrome (TIS) will be considered for inclusion. Categories of TIS include:
Key exclusion criteria (1 listed)
- Failure to meet inclusion criteria.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Synthes VEPTR (Vertical Expandable Prosthetic Titanium Rib) | Device | Expansion Thoracoplasty |
Primary outcome
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.)
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.)
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.)
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
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.)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 May 2020.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jeffrey Martus, MD principal investigator
Vanderbilt University Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Thoracic Insufficiency Syndrome (TIS), run by Vanderbilt University Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 May 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome 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.)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 May 2020.