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Diaphragm Pacing System (DPS) In Participants With Amyotrophic Lateral Sclerosis (ALS) (DPS in ALS)

Primary Purpose

Amyotrophic Lateral Sclerosis (ALS)

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
NeuRx® Diaphragm Pacing System™ (DPS)
Sponsored by
Barrow Neurological Institute
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Amyotrophic Lateral Sclerosis (ALS) focused on measuring ALS, DPS, Diaphragm Pacing

Eligibility Criteria

21 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Age 21 years or older.
  2. Sporadic or familial ALS diagnosed as definite, probable or possible ALS as defined by revised El Escorial criteria.

  3. Evidence of hypoventilation at Screening with at least one of the following:

    1. Maximal static inspiratory pressure (MIP) <60 cm H20.
    2. Upright or supine forced vital capacity (FVC) <50% predicted for gender, age, and height.
  4. A phrenic nerve potential should be recordable bilaterally.
  5. Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to randomization (riluzole-naïve subjects are permitted in the study).
  6. Capable of providing informed consent and following trial procedures.
  7. Geographically accessible to the site.
  8. Negative urine pregnancy test at Screening in women of child bearing potential (WOCBP). (Women who are post-menopausal or who have had a hysterectomy are deemed not of child bearing potential).
  9. Women of child bearing potential must use an adequate form of contraception: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal (patch or contraceptive ring, for example) contraception), intrauterine device (IUD) in place for ≥ 3 months, barrier method in conjunction with spermicide, or another adequate method.

Exclusion Criteria:

  1. Upright forced vital capacity (FVC) ≤ 45% of predicted for gender, age, and height.
  2. Any prior use of non-invasive ventilation (NIV) outside of sleep (nighttime or naps).
  3. Any pulmonary or cardiac disorder or other medical disorder that would be a contraindication for general anesthesia or DPS hardware implantation in the chest.
  4. Implanted electrical device such as a pacemaker or cardiac defibrillator.
  5. Known diaphragm abnormality such as hiatal hernia or para-esophageal hernia of abdominal contents into the thoracic cavity.
  6. Participation in another treatment research study for people with ALS.
  7. Exposure to any other agent currently under investigation for the treatment of people with ALS (off-label use or investigational) within 30 days of the Screening Visit.
  8. Clinically significant history of unstable or severe cardiac, oncologic, hepatic, psychiatric, renal disease, or other medically significant illness.
  9. Pregnant women or women currently breastfeeding.

Sites / Locations

  • Barrow Neurological Institute
  • Cedars-Sinai Medical Center
  • California Pacific Medical Center
  • Stanford University
  • Hospital for Special Care
  • Mayo Clinic Florida
  • University of Florida, Jacksonville
  • University of Iowa
  • Massachusetts General Hospital
  • Henry Ford Health Systems
  • St Louis University
  • Neurology Associates P.C.
  • Hospital for Special Surgery
  • SUNY Upstate Medical University
  • Carolinas Health Care
  • Wake Forest University
  • Cleveland Clinic
  • Ohio State University
  • Providence ALS Center
  • Drexel University
  • University of Pittsburgh
  • University of Texas Southwestern
  • University of Texas
  • University of Vermont
  • Virginia Mason Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

NeuRx® Diaphragm Pacing System™ (DPS)

Standard of Care

Arm Description

Patients randomized to the experimental arm will receive The NeuRx® Diaphragm Pacing System™ (DPS) device. Under general anesthesia, the intramuscular electrodes are surgically implanted in the diaphragm.

patients randomized to the standard of care arm will not have the Diaphragm Pacing System surgically implanted but will receive standard medical care.

Outcomes

Primary Outcome Measures

Survival

Secondary Outcome Measures

quality of life

Full Information

First Posted
August 23, 2013
Last Updated
March 7, 2023
Sponsor
Barrow Neurological Institute
Collaborators
ALS Association, Muscular Dystrophy Association, Synapse Biomedical
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1. Study Identification

Unique Protocol Identification Number
NCT01938495
Brief Title
Diaphragm Pacing System (DPS) In Participants With Amyotrophic Lateral Sclerosis (ALS)
Acronym
DPS in ALS
Official Title
Multi-Center, Randomized Controlled Study of the NeuRx® Diaphragm Pacing System™ (DPS)In Participants With Amyotrophic Lateral Sclerosis (ALS)
Study Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Completed
Study Start Date
August 2013 (undefined)
Primary Completion Date
January 2017 (Actual)
Study Completion Date
April 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Barrow Neurological Institute
Collaborators
ALS Association, Muscular Dystrophy Association, Synapse Biomedical

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The study is being conducted to determine if DPS treatment for people with ALS and hypoventilation is associated with improved survival or diaphragm function. The primary objective of the study is to conduct a multi-center, randomized controlled clinical trial comparing standard of care (control) to diaphragm stimulator treatment with the NeuRx® Diaphragm Pacing System™ (DPS) with respect to survival. The secondary objective of the study is to conduct a multi-center, randomized controlled clinical trial to compare standard of care treatment (control) to DPS in ALS subjects with hypoventilation.
Detailed Description
The study is a randomized controlled study to compare standard of care (control) to DPS (diaphragm stimulator named The NeuRx® Diaphragm Pacing System™). Although it remains uncertain if DPS is efficacious for the ALS subject, given the preliminary studies performed by the device manufacturer, it has received FDA humanitarian device exemption (HDE) approval as a humanitarian use device (HUD). This study will utilize a 2:1 randomization schedule such that eligible ALS subjects will have a 2/3 chance of receiving DPS and 1/3 chance of standard of care (control) treatment. The NeuRx® Diaphragm Pacing System™ (DPS) is a four channel, implanted percutaneous diaphragm muscle stimulation system. Under general anesthesia, the intramuscular electrodes are laparoscopically implanted in the diaphragm. The ends of the implanted electrodes are tunneled subcutaneously to an exit site on the chest or abdominal wall and connected to an external stimulator.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis (ALS)
Keywords
ALS, DPS, Diaphragm Pacing

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
52 (Actual)

8. Arms, Groups, and Interventions

Arm Title
NeuRx® Diaphragm Pacing System™ (DPS)
Arm Type
Experimental
Arm Description
Patients randomized to the experimental arm will receive The NeuRx® Diaphragm Pacing System™ (DPS) device. Under general anesthesia, the intramuscular electrodes are surgically implanted in the diaphragm.
Arm Title
Standard of Care
Arm Type
No Intervention
Arm Description
patients randomized to the standard of care arm will not have the Diaphragm Pacing System surgically implanted but will receive standard medical care.
Intervention Type
Device
Intervention Name(s)
NeuRx® Diaphragm Pacing System™ (DPS)
Intervention Description
The NeuRx® Diaphragm Pacing System™ (DPS) is a four channel, implanted percutaneous diaphragm muscle stimulation system. Pictures of the device are provided in the Clinician's Manual for the NeuRx® DPS procedure and technique guide from the manufacture Synapse Biomedical, Inc. Under general anesthesia, the intramuscular electrodes are laparoscopically implanted in the diaphragm. The ends of the implanted electrodes are tunneled subcutaneously to an exit site on the chest or abdominal wall and connected to an external stimulator.
Primary Outcome Measure Information:
Title
Survival
Time Frame
Subjects will be assessed on a monthly basis for an 18 month period.
Secondary Outcome Measure Information:
Title
quality of life
Time Frame
Subjects will be assessed on a monthly basis for an 18 month period.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Age 21 years or older. Sporadic or familial ALS diagnosed as definite, probable or possible ALS as defined by revised El Escorial criteria. Evidence of hypoventilation at Screening with at least one of the following: Maximal static inspiratory pressure (MIP) <60 cm H20. Upright or supine forced vital capacity (FVC) <50% predicted for gender, age, and height. A phrenic nerve potential should be recordable bilaterally. Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to randomization (riluzole-naïve subjects are permitted in the study). Capable of providing informed consent and following trial procedures. Geographically accessible to the site. Negative urine pregnancy test at Screening in women of child bearing potential (WOCBP). (Women who are post-menopausal or who have had a hysterectomy are deemed not of child bearing potential). Women of child bearing potential must use an adequate form of contraception: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal (patch or contraceptive ring, for example) contraception), intrauterine device (IUD) in place for ≥ 3 months, barrier method in conjunction with spermicide, or another adequate method. Exclusion Criteria: Upright forced vital capacity (FVC) ≤ 45% of predicted for gender, age, and height. Any prior use of non-invasive ventilation (NIV) outside of sleep (nighttime or naps). Any pulmonary or cardiac disorder or other medical disorder that would be a contraindication for general anesthesia or DPS hardware implantation in the chest. Implanted electrical device such as a pacemaker or cardiac defibrillator. Known diaphragm abnormality such as hiatal hernia or para-esophageal hernia of abdominal contents into the thoracic cavity. Participation in another treatment research study for people with ALS. Exposure to any other agent currently under investigation for the treatment of people with ALS (off-label use or investigational) within 30 days of the Screening Visit. Clinically significant history of unstable or severe cardiac, oncologic, hepatic, psychiatric, renal disease, or other medically significant illness. Pregnant women or women currently breastfeeding.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jeremy Shefner, MD, PhD
Organizational Affiliation
Barrow Neurological Institute
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Jonathan Katz, MD
Organizational Affiliation
California Pacific Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Barrow Neurological Institute
City
Phoenix
State/Province
Arizona
Country
United States
Facility Name
Cedars-Sinai Medical Center
City
Los Angeles
State/Province
California
Country
United States
Facility Name
California Pacific Medical Center
City
San Diego
State/Province
California
Country
United States
Facility Name
Stanford University
City
Stanford
State/Province
California
Country
United States
Facility Name
Hospital for Special Care
City
New Britain
State/Province
Connecticut
Country
United States
Facility Name
Mayo Clinic Florida
City
Jacksonville
State/Province
Florida
Country
United States
Facility Name
University of Florida, Jacksonville
City
Jacksonville
State/Province
Florida
Country
United States
Facility Name
University of Iowa
City
Iowa City
State/Province
Iowa
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
Country
United States
Facility Name
Henry Ford Health Systems
City
Detroit
State/Province
Michigan
Country
United States
Facility Name
St Louis University
City
Saint Louis
State/Province
Missouri
Country
United States
Facility Name
Neurology Associates P.C.
City
Lincoln
State/Province
Nebraska
Country
United States
Facility Name
Hospital for Special Surgery
City
New York
State/Province
New York
Country
United States
Facility Name
SUNY Upstate Medical University
City
Syracuse
State/Province
New York
Country
United States
Facility Name
Carolinas Health Care
City
Charlotte
State/Province
North Carolina
Country
United States
Facility Name
Wake Forest University
City
Winston-Salem
State/Province
North Carolina
Country
United States
Facility Name
Cleveland Clinic
City
Cleveland
State/Province
Ohio
Country
United States
Facility Name
Ohio State University
City
Columbus
State/Province
Ohio
Country
United States
Facility Name
Providence ALS Center
City
Portland
State/Province
Oregon
Country
United States
Facility Name
Drexel University
City
Philadelphia
State/Province
Pennsylvania
Country
United States
Facility Name
University of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
Country
United States
Facility Name
University of Texas Southwestern
City
Dallas
State/Province
Texas
Country
United States
Facility Name
University of Texas
City
San Antonio
State/Province
Texas
Country
United States
Facility Name
University of Vermont
City
Burlington
State/Province
Vermont
Country
United States
Facility Name
Virginia Mason Medical Center
City
Seattle
State/Province
Washington
Country
United States

12. IPD Sharing Statement

Links:
URL
http://alsconsortium.org
Description
Related Info

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Diaphragm Pacing System (DPS) In Participants With Amyotrophic Lateral Sclerosis (ALS)

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