CSP #2003 - Exoskeleton Assisted-Walking in Persons With SCI: Impact on Quality of Life ((PEPSCI))

• Mental Health Component Summary (MCS) of the Veterans Rand-36 (VR-36) [ Time Frame: Change from Baseline Assessment to 4 Months Post Intervention ]
  The Veterans RAND 36 Item Health Survey (VR-36) is a brief, generic, multi-use, self-administered health survey comprised of 36 items. The instrument is primarily used to measure health related quality of life, to estimate disease burden and to evaluate disease-specific impact on general and selected populations. The items on the questionnaire correspond to eight principal health domains including general health perceptions, physical functioning, role limitations due to physical and emotional problems, bodily pain, energy-fatigue, social functioning and mental health. The outcome in this study is the score from the Mental Health Component Summary (MCS) of the VR-36, which measures the vitality, social functioning, role-emotional, and mental health of the participant.
  
  
• The sum T-score for the SCI-QOL Physical Medical Health domain (three item banks of Bladder Management Difficulties, Bowel Management Difficulties, and Pain Interference) [ Time Frame: Baseline, Post Observation/Training phase, post 2 month Intervention, post 4 month Intervention ]
  The Primary Objectives are to demonstrate that Veterans with chronic SCI of =six months duration who are medically stable and are wheelchair users for indoor and outdoor mobility as their standard of care (SOC) plus use of an exoskeletal-assisted walking device in their home and community environments will have clinically meaningful net improvements in the MCS/VR-36 and in patient-reported outcomes for the SCI-QOL bladder, bowel, and pain item banks compared with those who use only SOC for home and community mobility. The primary outcomes to be assessed will be the MCS value and the sum T-score of the SCI-QOL bladder management difficulties, bowel management difficulties and pain interference item banks.
  
  

Background Veterans with spinal cord injury (SCI) have many adverse secondary medical and quality of life (QOL) changes as a result of immobilization. Veterans with SCI who have completed rehabilitation after injury and are unable to ambulate receive a wheelchair as standard of care (SOC) for mobility. Powered exoskeletons are a technology that has recently become available as an alternate form of mobility by providing an external framework for support and computer controlled motorized hip and knee joints to assist with overground ambulation.

Research Questions (Objectives) Will Veterans with chronic SCI of six months duration, who are medically stable and who use a wheelchair as SOC plus an exoskeletal-assisted walking (EAW) device in their home and community environments have clinically meaningful net improvements in mental health, bladder, bowel, and pain patient-reported outcomes compared with those who use only the SOC? Additionally, will the use of an EAW device for four months in the homes and/or communities of the participants result in a reduction of total body fat mass? Study Design A two-group (Intervention and Control), randomized, clinical trial will be performed with a one-year feasibility component. The Intervention group will receive SOC plus EAW. The Control group will receive SOC only. The study will require six years in total to complete and includes ten VA SCI Services as study sites. A feasibility phase will be employed using a staggered start by initially starting six sites and the other four sites starting one year later. These initial six sites will be used to assess the start-up activities [hiring, training, equipment procurement, Central Institutional Review Board (CIRB) paperwork, etc.], the rate of recruitment, and any other issues that may be of value for the successful completion of the study. Lessons learned will be implemented for the remaining sites.

Relevance to VA In pilot studies conducted at the James J. Peters VA Medical Center, Bronx, NY, improvements in mental-emotional health, physical health and body composition were demonstrated by providing the participants the ability to walk for 4 to 6 hours per week over the course of three to five months. As of July 2014, a Class 2 designation was established by the Food and Drug Administration (FDA) for "powered exoskeletons". To date, one device (ReWalk ) has received FDA Class 2 approval for institutional and home use and is currently available by prescription. The Department of Veterans Affairs (DVA) is the largest single provider to persons with SCI in the USA, caring for about 26,000 of the 42,000 estimated Veterans with SCI. The VA presently lacks the infrastructure to support Veterans with SCI to train to use this device in order to make this technology available for the home/community use. A controlled research study would be anticipated to be the optimal manner to demonstrate the efficacy, amount of use and safety of a powered exoskeleton in the home and community environments; findings would be immediately transferable to clinical care.

Number of Research Participants (Sample Size) One hundred-sixty participants (80/group) will be randomized. Each of the 10 study sites will be expected to pre-screen 100 potential participants, screen 60 participants, and randomize 16 participants (8 per group) over the 2-year enrollment period.

Participating Sites Ten SCI Services will be selected on the basis of potential recruitment numbers (N=9,511 total Veterans with SCI in the sites' catchment areas and N=4,921 followed annually at these sites) and geographic location, to permit an even distribution across the country. The ten sites include: Boston, Richmond, St. Louis, Tampa, Milwaukee, Minneapolis, Dallas, Houston, Palo Alto, and Long Beach. Of these ten sites, five are VA Cooperative Studies Program (CSP) Network of Dedicated Enrollment Sites (NODES).

Duration of Participant Intake (Study Duration) The CS #2003 study duration is projected to be a total of six years: The initial six sites will have a start-up year, followed by participant enrollment/data collection during years 1 and 2, and continued data collection/closeout during year 3, for a total of four years. The remaining four sites will begin the start-up year one year after the first six sites and follow the same enrollment, data collection and closeout schedule over the next four years. The four sites are expected to be completed one year after the initial six sites have completed, and there is an additional year for the Coordinating Center and Chairperson's Office to complete data analysis and manuscript writing, thus the study total time is six years. Participants in both groups will be asked to commit 33 weeks to this study. Participants in the Control arm will be offered an additional 8 weeks to receive EAW training in the medical centers, without outcome data being collected.

Treatment (follow-up) The intervention being tested is four months of home and/or community use of a powered exoskeleton.

Definition of Participant Samples (Study Population) One-hundred sixty male or female Veterans / Active Duty military personnel with chronic SCI, six months duration, 21 years of age, functional use of their hands, medically stabile, and wheelchair users for indoor and outdoor mobility, will be eligible for screening. All potential participants will be Veterans / Active Duty military personnel with SCI. Study participants will generally be outpatients with the exception of those inpatients who meet the eligibility criteria, and who are approved by the Site Investigator (e.g., some inpatients may have been admitted for a wheelchair fitting or another non-medical reason). Non-veterans with SCI will not be.

Treatment Arms All participants will receive four months of treatment, randomized into two arms: SOC plus EAW or SOC only.

Endpoints Primary outcome one will be the Mental Component Summary of the Veterans Rand-36 (MCS/VR-36). Primary outcome two will be the sum T-score of the SCI-QOL bladder management difficulties, bowel management difficulties and pain interference item banks. The major secondary outcome will be total body fat mass. The two primary and the major secondary outcomes will be analyzed as the proportion of participants in each group who achieved a clinically meaningful change in score. The endpoint will be success or failure for these outcomes.