Pilot Clinical Trial will Test Diaphragm Stimulation Device
October 28, 2004
Raymond P. Onders, M.D. of the University Hospitals of Cleveland has informed The ALS Association about a new pilot study in ALS for which he is the principal investigator. Dr. Onders and his colleagues report that they are preparing to begin a clinical trial titled, Muscle Stimulation of the Diaphragm in ALS, in a small group of patients with ALS. The purpose of this research study is to find out if an experimental “diaphragm pacing system can provide stimulation to the diaphragm to maintain its strength and potentially help with the breathing problems associated with ALS.” Although there have been similar studies with encouraging results in a few people with spinal cord injuries, the diaphragm pacing system has not been tested in people living with ALS.
The study includes a surgical procedure to place electrodes (flat metal disks) onto the diaphragm. The electrodes will be attached to an electric stimulator device that is outside of the body. General anesthesia is required and the surgical procedure is done through a laparoscope. As with any surgery, and for people with ALS with weakened breathing muscles, there are risks associated with the procedure. Dr. Onders indicates that the study has been approved by the Food and Drug Administration (FDA) and an institutional review board (IRB). “This is an investigational study that we hope with time will lead us to helping patients with ALS be able to breathe better and have an improved quality of life,” states Dr. Onders.
The investigators have applied for grants to help cover the costs of this study, but at present this is an un-sponsored study. It is possible that some of the expenses may be covered by Medicare and Medicaid, and perhaps, private insurance. People interested in participating in the study should ask the investigators for a listing of all anticipated out-of-pocket expenses. The ALS Association will keep the ALS community informed about grants for this research project and the status of insurance coverage as more information is known.
This 12-month study includes nine visits to the University Hospitals of Cleveland and will enroll ten people with ALS who meet the inclusion criteria. There is an extensive initial assessment and screening process.
The primary questions this study is designed to answer are:
- Are the surgical procedure and electrical stimulation of the phrenic nerve safe in people with ALS?
- Is the electrical stimulation tolerable for people with ALS?
After the surgery, the nine months of the study include having the patient or caregiver gradually increase the power and frequency of the electric stimulator (pacing device) to “train” the diaphragm. In addition, the study will evaluate whether the electric stimulation of the phrenic nerve can cause diaphragm muscle contraction in a manner that will support breathing.
The study is not designed to slow or stop the progression of ALS, and some patients in the study may develop symptoms of respiratory muscle weakness and trouble breathing. People in the study, who need support for breathing such as noninvasive ventilation (BiPAP) at some point, should not delay seeking care.
Q and A:
Q: Is it unusual for a research study to require that the patients pay out-of-pocket?
A: Yes, it is unusual to have patients in a research study responsible for the costs of the study. This is a concern for The ALS Association; however, there is the possibility of grant sponsorship the investigators have applied for, and insurance companies may pay for a portion of the study expenses.
Q: Will the diaphragm pacing device support my breathing so that I will not need to use noninvasive ventilation (BiPAP) or long-term mechanical ventilation with a tracheostomy?
A: At this time, no one knows if the electrical stimulation will support breathing for people with ALS. Although the primary purpose of this study is to evaluate the safety and tolerability of the procedure and the electrical stimulation, the investigators will also evaluate if the device supports breathing and/or strengthens the diaphragm muscle.
Q: Was the diaphragm pacing device tested in an ALS animal model?
A: The ALS Association is not aware of any ALS animal model studies using this device and would be very interested in seeing such a study conducted. The questions the research investigators are asking in this study are important and could lead to a major advance in the care of people with ALS. However, there are several yet unanswered points such as how to evaluate outcomes without control groups, and the relative value of the pacing device compared to noninvasive ventilation from cost and quality-of-life perspectives.
Q: If my breathing tests are normal and I am not having any trouble breathing now, can I enroll in the study to prevent future muscle weakness?
A: No, for a person who does not have diaphragm muscle weakness, it is not appropriate to undergo the surgery and electric stimulation. To meet the inclusion criteria established by the investigators for this study, people must have evidence of diaphragm weakness within a specific range that has been determined by the study investigators.
Q: How can I get more information about this study?
A: Contact Dr. Raymond P. Onders by email at Raymond.Onders@uhhs.com
