NeuRx Diaphram Pacing System (DPS) Use in Amyotrophic Lateral Sclerosis (ALS)

Study Overview
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Brief Summary
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The purpose of this research study is to collect more information about the use, safety, and effectiveness of the NeuRx DPS® in ALS patients.

Detailed Description
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Characterize the safety and benefit of on-label Humanitarian Device Exemption (HDE) use of DPS in patients with ALS who initiated noninvasive ventilation (NIV).

Official Title
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Multi-Center, Concurrent-Controlled, On-Label Study of the NeuRx Diaphragm Pacing System (DPS) in Participants With Amyotrophic Lateral Sclerosis (ALS)

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Device: NeuRx DPS


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age 21 or older Familial or sporadic ALS diagnosed as possible, laboratory-supported probable, probable, or definite according to the World Federation of Neurology El Escorial criteria Bilateral phrenic nerve function clinically acceptable as demonstrated by bilateral diaphragm movement with fluoroscopic sniff test or with phrenic nerve conduction studies Chronic hypoventilation was documented by at least one of the following: MIP (respiratory muscle strength) less than 60 cmH2O, or Forced Vital Capacity (FVC) less than 50% predicted, or PaCO2 greater than or equal to 45 mmHg, or Nocturnal SaO2 (arterial oxygen saturation) less than or equal to 88% for at least five continuous minutes Initiation of non-invasive ventilation at or before time of enrollment Suitable surgical candidate to receive diaphragm pacing stimulation Negative pregnancy test in female participants of childbearing potential (treatment group) Informed consent from patient or designated representative Exclusion Criteria: FVC less than 45% predicted within 10 days prior to surgery. Participation in simultaneous therapeutic clinical trial which could affect the safety or benefit outcomes of this study.

Ages Eligible for Study
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Minimum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: NeuRx DPS<br>Subjects who have initiated noninvasive ventilation (NIV) and receive the NeuRx diaphragm pacing system (DPS). | Device: NeuRx DPS<br>* Patients who are diagnosed with ALS, have initiated NIV and choose to have the NeuRx DPS implanted.<br>* Other names: Diaphragm Pacing System;|
| No Intervention: No DPS<br>Subjects who have initiated noninvasive ventilation (NIV) and do not receive the DPS device. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Survival | Compare the survival of patients who initiated noninvasive ventilation (NIV).and who receive DPS to controls without DPS: (1) concurrent controls; (2) historical controls (e.g., Lechtzin and PRO-ACT databases); and (3) virtual controls (Origent). | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Relationship between survival time and clinical features of ALS | Determine whether there is a relationship between survival time and ALS onset type. | 24 months | 
| Relationship between survival time and ALS assessment scores | Determine whether there is a relationship between survival time and Upper Motor Neuron (UMN)/ Lower Motor Neuron (LMN) assessment scores. | 24 months | 
| Relationship between survival time and onset of weakness from ALS to treatment |  | 24 months | 
| Relationship between survival time and ALS treatment interventions |  | 24 months | 
| Relationship between survival time and intraoperative strength of contraction |  | 24 months | 
| Characterize change in overall and respiratory function | Characterize longitudinal change in overall function (ALSFRS-R) before and after treatment with DPS compared to controls. | 24 months | 
| Characterize change in respiratory function | Characterize longitudinal change in respiratory function (FVC, MIP) before and after treatment with DPS compared to controls. | 24 months | 
| Types device and procedure-related adverse events |  | 24 months | 
| Frequency of device and procedure-related adverse events |  | 24 months | 
| Types of respiratory serious adverse events |  | 24 months | 
| Frequency of respiratory adverse events |  | 24 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Diaphragm Pacing