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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by Synapse Biomedical
Sponsor:
Information provided by (Responsible Party):
Synapse Biomedical
ClinicalTrials.gov Identifier:
NCT01605006
First received: May 22, 2012
Last updated: May 30, 2014
Last verified: May 2014
  Purpose

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.


Condition Intervention
Amyotrophic Lateral Sclerosis (ALS)
Device: NeuRx Diaphragm Pacing System (DPS)

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: HDE Post-Approval Study (PAS) of NeuRx DPS for ALS

Resource links provided by NLM:


Further study details as provided by Synapse Biomedical:

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 ] [ Designated as safety issue: Yes ]
    • 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 ] [ Designated as safety issue: No ]
    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.)


Estimated Enrollment: 60
Study Start Date: July 2012
Estimated Study Completion Date: September 2017
Estimated Primary Completion Date: September 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
NeuRx DPS On-Label Treatment
All study participants who meet the study eligibility criteria will undergo the surgical implantation procedure to receive the NeuRx DPS. Participants who are successfully implanted with the electrodes will use the NeuRx DPS system for diaphragm conditioning.
Device: NeuRx Diaphragm Pacing System (DPS)
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.
Other Names:
  • diaphragm pacing
  • diaphragmatic pacing
  • phrenic nerve stimulation

Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   21 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age 21 or older.
  2. Participants with familial or sporadic ALS diagnosed as laboratory-supported probable, probable, or definite according to the World Federation of Neurology El Escorial criteria.
  3. Bilateral phrenic nerve function clinically acceptable as demonstrated by bilateral diaphragm movement with fluoroscopic sniff test or with EMG recordings and nerve conduction times.
  4. 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
  5. Suitable surgical candidate.
  6. Negative pregnancy test in female participants of childbearing potential.
  7. Informed consent from patient or designated representative.

Exclusion Criteria:

  1. Underlying cardiac or pulmonary disease that would increase the risk of general anesthesia.
  2. Underlying pulmonary diseases that were present prior to ALS that would affect pulmonary tests independent of ALS.
  3. Uncontrolled excessive secretions.
  4. FVC less than 45% predicted at time of surgery.
  5. Preexisting implanted electrical device such as pacemaker or cardiac defibrillator.
  6. Pre-existing diaphragm abnormality such as a hiatal hernia or paraesophageal hernia of abdominal contents going into the thoracic cavity.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01605006

Contacts
Contact: Michael Fritz 888-767-3770 ext 134 mfritz@synapsebiomedical.com

Locations
United States, California
Cedars-Sinai Medical Center Recruiting
Los Angeles, California, United States, 90048
Contact: Tami Kendra-Romito       tami.kendra@cshs.org   
Contact: Robert Baloh, MD       robert.baloh@csmc.edu   
Principal Investigator: Robert Baloh, MD         
California Pacific Medical Center -- Forbes Norris MDA/ALS Research Center Recruiting
San Francisco, California, United States, 94115
Contact: Robert G. Miller, M.D.    415-600-3604    MillerRX@cpmcri.org   
Principal Investigator: Robert G. Miller, M.D.         
United States, Colorado
University of Colorado Denver Recruiting
Denver, Colorado, United States, 80045
Contact: Alexander (AJ) Stein       alexander.stein@ucdenver.edu   
Contact: Teerin Liewluck, MD       teerin.liewluck@ucdenver.edu   
Principal Investigator: Teerin Liewluck, MD         
United States, Florida
Mayo Clinic Recruiting
Jacksonville, Florida, United States, 32224
Contact: Pamela DeSaro       desaro.pamela@mayo.edu   
Contact: Kevin Boylan, MD       boylan.kevin@mayo.edu   
Principal Investigator: Kevin Boylan, MD         
United States, Illinois
Northwestern University Recruiting
Chicago, Illinois, United States, 60611
Contact: Helen Donnelly       h-donnelly@northwestern.edu   
Contact: Lisa Wolfe, MD       lwolfe@northwestern.edu   
Principal Investigator: Lisa Wolfe, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Maureen Walsh       mwalsh@kumc.edu   
Contact: April McVey, MD       amcvey@kumc.edu   
Principal Investigator: April McVey, MD         
United States, Nebraska
University of Nebraska Medical Center Recruiting
Omaha, Nebraska, United States, 68198
Contact: Elspeth McKeon       emckeon@nebraskamed.com   
Contact: Jose Americo Fernandes Filho, MD       jfernandes@unmc.edu   
Principal Investigator: Jose Americo Fernandes Filho, MD         
United States, New York
Stony Brook University Recruiting
Stony Brook, New York, United States, 11794
Contact: Jessica Lamb       jessica.lamb@stonybrookmedicine.edu   
Contact: Nurcan Gursoy, MD       nurcan.gursoy@stonybrookmedicine.edu   
Principal Investigator: Nurcan Gursoy, MD         
United States, North Carolina
Duke University Recruiting
Durham, North Carolina, United States, 27705
Contact: Karen Grace       karen.grace@duke.edu   
Contact: Richard Bedlack, MD       richard.bedlack@duke.edu   
Principal Investigator: Richard Bedlack, MD         
United States, Ohio
University Hospitals Case Medical Center Recruiting
Cleveland, Ohio, United States, 44106
Contact: Mary Jo Elmo       maryjo.elmo@uhhospitals.org   
Contact: Raymond Onders, MD       raymond.onders@uhhospitals.org   
Principal Investigator: Mary Jo Elmo, CNP         
United States, Oregon
Providence St. Vincent Medical Center Recruiting
Portland, Oregon, United States, 97213
Contact: Nancy Hoke       nancy.hoke@providence.org   
Contact: Kimberly Goslin, MD       kimberly.goslin@providence.org   
Principal Investigator: Kimberly Goslin, MD         
Sponsors and Collaborators
Synapse Biomedical
Investigators
Principal Investigator: Robert G. Miller, M.D. Forbes Norris MDA/ALS Research Center, California Pacific Medical Center
  More Information

Additional Information:
No publications provided

Responsible Party: Synapse Biomedical
ClinicalTrials.gov Identifier: NCT01605006     History of Changes
Other Study ID Numbers: CLIN 20-0009-0020
Study First Received: May 22, 2012
Last Updated: May 30, 2014
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by Synapse Biomedical:
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

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Sclerosis
Motor Neuron Disease
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurodegenerative Diseases
TDP-43 Proteinopathies
Neuromuscular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on August 18, 2014