Humanitarian Device Exemption Post-Approval Study of NeuRx Diaphragm Pacing System for Amyotrophic Lateral Sclerosis

Primary Outcome Measures:

• Safety Outcome Measure -- Occurrence of major device-related (including procedure-related) adverse events as defined below. [ Time Frame: follow-up assessments at 3-month intervals ]
  • Serious capnothorax requiring invasive intervention
  • Mechanical ventilation for 24 hours or longer post-procedure
  • Post-procedure extubation failure resulting in permanent tracheostomy ventilation
  • Perioperative complication which delays initiation of NeuRx DPS therapy
  • Severe discomfort due to stimulation which is unable to be tolerated or resolved
  • Device malfunction which interrupts or causes an undesired diminution of NeuRx DPS therapy
  • Electrode dislodgement from the diaphragm
  • Wire infection
  • Any other device- or procedure-related serious adverse event
  
  

Secondary Outcome Measures:

• Probable Benefit Outcome Measure [ Time Frame: follow-up assessments at 3-month intervals ]
  Survival, defined as time to (a) death or (b) permanent tracheostomy mechanical ventilation (PTV) with discontinuation of pacing. (All deaths and PTV events will be reported regardless of relationship to the device or procedure.)
  
  

This is a prospective, non-randomized, open-label, interventional, post-approval (FDA) study of the NeuRx Diaphragm Pacing System (DPS) device. The study will enroll 60 participants who have amyotrophic lateral sclerosis (ALS), meet the FDA-approved device indications for use, and undergo the surgical implantation procedure to receive the device. The device is intended for use in ALS patients with a stimulatable diaphragm (both right and left portions) as demonstrated by voluntary contraction or phrenic nerve conduction studies, and who are experiencing chronic hypoventilation (CH), but not progressed to an FVC less than 45% predicted. Participants who are successfully implanted with the device will use it for daily diaphragm conditioning sessions. Participants will be followed for at least two years (until the last enrolled participant reaches the 2-year follow-up visit). Safety and probable benefit outcome measures will be assessed. The primary objective of the study is: (1) (Safety) Characterize the types and frequency of major device-related adverse events (AEs) over the time of device use. Secondary objectives of the study are: (2) (Safety) Determine whether the frequency of major device-related AEs increases dramatically toward end of life; and (3) (Probable Benefit) Determine whether there is a relationship between survival time and onset type (bulbar and limb), time from onset to treatment, and use of NIV, riluzole, or PEG in patients treated with the device.