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

NCT01605006 | Completed

• 1 other identifier: CLIN 20-0009-0020
• Study Type: interventional
• Target: 97
• Locations: 1 country
• Sites: 11

Brief Summary

This post-approval study will follow 60 participants who have ALS, documented chronic hypoventilation, and bilateral phrenic nerve function, and who undergo the surgical implantation procedure to receive the NeuRx Diaphragm Pacing System device. 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.

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Keywords

amyotrophic lateral sclerosis; ALS; motor neuron disease; diaphragm; diaphragm pacing; diaphragmatic pacing; phrenic pacing; phrenic nerve; phrenic nerve stimulation; NeuRx DPS; DPS; chronic hypoventilation; respiration; ventilation; breathing; Humanitarian Device Exemption; HDE; post-approval study

Outcome Measures

Primary Outcomes (1)

• Safety Outcome Measure -- Occurrence of major device-related (including procedure-related) adverse events as defined below.

  * 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

  follow-up assessments at 3-month intervals

Secondary Outcomes (1)

• Probable Benefit Outcome Measure

  follow-up assessments at 3-month intervals

Study Arms (1)

NeuRx Diaphragm Pacing System (DPS)

OTHER

Surgical implantation of the NeuRx DPS (on label use).

Device: NeuRx Diaphragm Pacing System (DPS)

Interventions

NeuRx Diaphragm Pacing System (DPS) DEVICE

The NeuRx DPS is a percutaneous, intramuscular, diaphragm stimulation system which is implanted using standard laparoscopic surgical techniques in an outpatient procedure. The implanted intramuscular diaphragm electrodes are connected to a four channel external stimulator at a percutaneous exit site. DPS is designed to help ALS patients breathe by providing conditioning stimulation of the diaphragm muscles. Recommended frequency of diaphragm conditioning is at least 3 times per day with each session lasting at least 30 minutes. Patients may find it helpful to use the DPS for longer periods to help with breathing. DPS may be used at the same time as non-invasive ventilation. Patents may also sleep with the DPS to assist with breathing difficulties at night.

Also known as: diaphragm pacing, diaphragmatic pacing, phrenic nerve stimulation

NeuRx Diaphragm Pacing System (DPS)

Eligibility Criteria

• Age: 21 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 21 or older.
• Participants with familial or sporadic ALS diagnosed as 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 EMG recordings and nerve conduction times.
• Chronic hypoventilation was documented by at least one of the following:
• FVC less than 50% predicted, or
• \|MIP\| less than 60 cmH2O, or
• PaCO2 greater than or equal to 45 mmHg, or
• Nocturnal SaO2 less than or equal to 88% for at least five continuous minutes
• Suitable surgical candidate.
• Negative pregnancy test in female participants of childbearing potential.
• Informed consent from patient or designated representative.

You may not qualify if:

• Underlying cardiac or pulmonary disease that would increase the risk of general anesthesia.
• Underlying pulmonary diseases that were present prior to ALS that would affect pulmonary tests independent of ALS.
• Uncontrolled excessive secretions.
• FVC less than 45% predicted at time of surgery.
• Preexisting implanted electrical device such as pacemaker or cardiac defibrillator.
• Pre-existing diaphragm abnormality such as a hiatal hernia or paraesophageal hernia of abdominal contents going into the thoracic cavity.

Sponsors & Collaborators

• Synapse Biomedical: lead

Study Sites (11)

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

California Pacific Medical Center -- Forbes Norris MDA/ALS Research Center

San Francisco, California, 94115, United States

Location

University of Colorado Denver

Denver, Colorado, 80045, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

Stony Brook University

Stony Brook, New York, 11794, United States

Location

Duke University

Durham, North Carolina, 27705, United States

Location

University Hospitals Case Medical Center

Cleveland, Ohio, 44106, United States

Location

Providence St. Vincent Medical Center

Portland, Oregon, 97213, United States

Location

Related Links

• FDA Summary of Safety and Probable Benefit for Humanitarian Device Exemption (Approved September 28, 2011)

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Motor Neuron Disease; Respiratory Aspiration

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Respiration Disorders; Respiratory Tract Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Robert G. Miller, M.D.

  Forbes Norris MDA/ALS Research Center, California Pacific Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 22, 2012
• First Posted: May 24, 2012
• Study Start: July 1, 2012
• Primary Completion: February 1, 2017
• Study Completion: February 1, 2017
• Last Updated: March 27, 2020

Record last verified: 2020-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (11)