The Big Idea: Your top questions answered

Posted by Reeve Staff in Daily Dose on December 15, 2017 # Research, News
1) What does FDA approval mean for The Big Idea and what are the next steps?

FDA approval for The Big Idea study indicates that we have the green light to begin the research and start to screen and enroll participants.

Researchers at the University of Louisville have implanted a total of 12 individuals living with chronic complete spinal cord injury. With each participant, new information and insights have strengthened researchers’ understanding of epidural stimulation and have influenced the structure of The Big Idea study.

Researchers are actively screening participants based on FDA-approved criteria. All candidates are being sourced from the University of Louisville’s database, Victory Over Paralysis. Please visit https://victoryoverparalysis.org/participate-in-re... if you are interested in registering your information to participate in The Big Idea and/or other clinical research studies being conducted at the University of Louisville.

2) How can I participate in The Big Idea? How can an individual be selected for The Big Idea? Can someone still enroll?

Anyone interested in participating in The Big Idea should register their information in the University of Louisville’s research database: https://victoryoverparalysis.org/participate-in-re...

It is important to note that the database is not just for The Big Idea – it is a resource for all spinal cord clinical studies at the University of Louisville’s Kentucky Spinal Cord Injury Research Center.

The Reeve Foundation does not have access to the KSCIRC database and does not know the status of individuals who have submitted their names as potential research candidates. A team of researchers led by Dr. Susan Harkema have complete responsibility for selecting participants and conducting the study.

If you match the FDA-approved criteria, you will be notified. Unfortunately, it is not possible for researchers to contact every person who has indicated their interest in becoming one of the 36 research participants for The Big Idea.

3) Where will this study take place? Are individuals from other countries eligible to participate?

The Big Idea is being conducted at the University of Louisville in Kentucky. Individuals from outside the United States are eligible to participate provided that they meet the study criteria and travel requirements. All interested candidates should register their information with the University of Louisville research database: https://victoryoverparalysis.org/participate-in-re...

4) What are researchers looking for when screening participants?

Please visit clinicaltrials.gov (NCT03364660) for a full overview of the study protocol and inclusion/exclusion criteria that was approved by the FDA.

Researchers from the University of Louisville are conducting the study and will decide which candidates meet the study criteria. Inclusion criteria are characteristics that candidates must have if they are to be included in the study; exclusion criteria are those characteristics that disqualify prospective subjects from being in the study.

5) What is the goal for The Big Idea?

The Big Idea will further explore the life-enhancing effects of epidural stimulation on people with spinal cord injury (SCI).

This research will demonstrate whether epidural stimulation will promote improved autonomic function (in particular, cardiovascular recovery) and the ability to stand and to voluntarily control leg movements below the injury level. The Big Idea will include 36 individuals living with chronic complete spinal cord injury.

Researchers led by principal investigator Dr. Susan Harkema at the University of Louisville’s Kentucky Spinal Cord Injury Research Center are seeking to demonstrate the safety and efficacy of epidural stimulation as a treatment for SCI, so as to potentially expedite its availability to individuals who can benefit from it. The outcomes of The Big Idea will determine the next steps required to translate the intervention to the clinic.

The Big Idea continues groundbreaking epidural stimulation research funded by the Reeve Foundation and published in 2014 in the scientific journal Brain. In that study, four young men diagnosed with chronic complete spinal cord injury were implanted with a device called an epidural stimulator on their spine. The men regained the ability to stand, bear their weight, and flex their toes, legs, and hips. They have also experienced improved autonomic functions, including bladder, bowel and sexual function.

6) Will I have any expenses if I agree to participate (travel/lodging)?

Individuals selected for The Big Idea will be reimbursed for their mileage traveling to and from Louisville, KY, throughout the duration of their two-year participation in the study. However, they will be responsible for their own food and lodging during their visits.

7) What is The Big Idea timeline going forward now that there is FDA approval? Can we expect periodic updates?

Now that we have obtained approval from the FDA, we can begin to screen and enroll participants in The Big Idea study. FDA approval is a significant milestone as it is the official green light to start this research. In total, 36 participants will be enrolled into The Big Idea. Each participant will remain in the study for two-years from enrollment through final discharge.

It will take a total of six years to enroll and discharge all 36 study participants as researchers will be closely monitoring the impact of epidural stimulation on each individual for experimental testing, data collection and observation.

Since The Big Idea is research involving human subjects, we are required to be careful and sensitive about what information can be shared publicly. However, the Foundation will work closely with researchers so that we can keep our community and other interested parties updated on progress.

8) After the trial, will participants be able to keep the implanted stimulator?

Research participants will be given the option to keep the implanted stimulator at the conclusion of their two-year participation. However, those who decide to keep the stimulator will be mandated to follow-up with the University of Louisville periodically.

9) What’s the difference between The Big Idea and the other epidural stimulation studies being advertised abroad?

The Big Idea will enroll subjects and conduct a series of experiments according to an FDA-approved study protocol.

The Christopher & Dana Reeve Foundation is aware that there are companies and clinics in other parts of the world that offer epidural stimulation alone or in combination with other interventions (i.e. stem cell transplants). Narratives, advertisements and patient testimonials on their websites and in their social media suggest that their "therapies" are the equivalent of the epidural stimulation studies being done at the University of Louisville. These medical treatments are not the same.

The Big Idea has US Food and Drug Administration (FDA) approval for a very specific research protocol and patient population. The protocol is the study plan that dictates how the research will be conducted from screening participants to collecting and disseminating data.

The Big Idea builds on several decades of research and will require a specific protocol in order to meet the safety and effectiveness requirements needed to gain FDA approval for epidural stimulation as a therapy for spinal cord injury.

While we received approval to being the study, epidural stimulation is not FDA-approved as a therapy for spinal cord injury and is still considered experimental. Only after safety and efficacy is rigorously demonstrated will the FDA approve its use for spinal cord injury. That is what the Reeve Foundation and the University of Louisville are working to achieve.

Note: There are companies and clinics in other parts of the world that are falsely advertising epidural stimulation treatments and may pose a danger to the paralysis community. Please read the Reeve Foundation's statement on off-shore stimulation treatments.