In 2011, the NeuRx® Diaphragm Pacing System (DPS) was granted Humanitarian Device Exemption (HDE H100006) approval by the United States Food and Drug Administration (FDA) for treating chronic hypoventilation from ALS. The decision was based on a multicenter clinical trial that enrolled 106 people with ALS and treated 86 for chronic hypoventilation. As a result of the FDA approval, the NeuRx® treatment is no longer experimental and is covered by commercial and government insurance.
In 2012 Synapse Biomedical, the ALS Association and the Muscular Dystrophy Association jointly committed to fund an expanded diaphragm pacing clinical trial, NCT01938495. Synapse Biomedical is supporting the follow on study to expand the body of evidence regarding the therapeutic effect of conditioning the diaphragm muscle on dyspnea (shortness of breath), sleep, quality of life over time and delaying the need for invasive ventilation.
The trial is led by co-principal investigators Jonathan Katz M.D., director of the neuromuscular diseases program at California Pacific Medical Center in San Francisco and Jeremy Shefner, M.D., Chair of the Department of Neurology and Associate Director for Barrow Neurological Institute. The trial of 180 people with ALS, will randomize 120 participants to receive the diaphragm pacing treatment in addition to other standard-of-care respiratory treatment (e.g., NIV), while 60 will be randomized into the control group and receive the current standard of care without diaphragm pacing. Once in the DPS control group a participant would be ineligible for other ALS study opportunities. Also, unlike a drug study where the control group members and their physicians are blinded to whether they receive the treatment drug or a placebo, the DPS participant in the device study are aware of which group they have been randomized. Additionally, unlike most drug studies, where access to the drug being tested is not available outside the study, the DPS ALS treatment is commercially available. You should be aware that you do not need to enroll in this randomized study (or any other study such as the company-sponsored post-approval study) to access the DPS treatment.
Despite the general HDE approval granted by FDA, the following clinical trial centers have restricted their prescription of the device to participants in the study. At this time, they are not offering the surgical treatment outside of the randomized clinical trial.