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

Not Applicable

Withdrawn

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Device: NeuRx DPS

• Registration Number: NCT03034317
• Lead Sponsor: Synapse Biomedical

Brief Summary

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

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

Study Timeline

• Study First Submitted: 2017-01-13
• Study First Submitted QC: 2017-01-25
• Study First Posted: 2017-01-27
• Study Start: 2017-02-02
• Primary Completion: 2018-02-08
• Study Completion: 2018-02-12
• Status Verified: 2020-03
• Last Update Submitted: 2020-03-24
• Last Update Posted: 2020-03-26

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
NeuRx DPS	NeuRx DPS	Subjects who have initiated noninvasive ventilation (NIV) and receive the NeuRx diaphragm pacing system (DPS).

Primary Outcome Measures

Name	Time	Method
Survival	24 months	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).

Secondary Outcome Measures

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