Multi-Center, Randomized Controlled Study of the NeuRx® Diaphragm Pacing System™ (DPS) In Participants with Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

The purpose of this study is to determine if surgical implantation of the NeuRx® Diaphragm Pacing System™ (DPS) improves survival or diaphragm function in individuals with ALS and breathing muscle weakness.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Device

Study Status:

Closed

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Jeremy Shefner, MD (Barrow Neurological Institute)
Jonathan Katz, MD (California Pacific Medical Center)

Clinicaltrials.gov ID (11 digit #):

NCT01938495

Neals Affiliated?

Yes

Coordinating Center Contact Information

Massachusetts General Hospital
Daniela Grasso / .(JavaScript must be enabled to view this email address) / 617-726-0842
.(JavaScript must be enabled to view this email address) 165 Cambridge Street
Floor 6
Boston, Massachusetts 02114 United States

Full Study Summary:

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder affecting primarily motor neurons, for which treatment designed to slow or arrest progression remains lacking. As the disease progresses, breathing can become difficult due to weakening of the breathing muscles. The NeuRx® Diaphragm Pacing System™ (DPS) is a four channel, implanted percutaneous diaphragm muscle stimulation system, used to assist in diaphragm functioning. Since DPS has not been rigorously evaluated in ALS populations, a clinical trial has been designed to scientifically determine the effectiveness on improvement of diaphragm function and survival in ALS patients.

Approximately 180 eligible subjects with ALS will be recruited from multiple centers in the US. Subjects will be randomized 2:1 to receive the DPS implant or standard of care (control).

Study Sponsor:

Muscular Dystrophy Association (MDA), The ALS Association, and Synapse Biomedical, Inc.

Participant Duration:

Subjects will take part in the study for approximately 18 months. Subjects will have 6 in-person visits and monthly phone visits during the study.

Estimated Enrollment:

52

Estimated Study Start Date:

07/31/2013

Estimated Study Completion Date:

04/30/2017

Posting Last Modified Date:

08/25/2017

Date Study Added to alsconsortium.org:

11/02/2016
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    21

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Study subjects meeting all of the following criteria will be allowed to enroll in the study:
    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:
    a. Maximal static inspiratory pressure (MIP) <60 cm H20
    b. Upright or supine forced vital capacity (FVC) <50% predicted for gender, age, and height
    4. Both hemi-diaphragms must be able to be stimulated with standard phrenic nerve conduction studies
    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
    Study subjects meeting any of the following criteria during screening evaluations will be excluded from entry into the study:
    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 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.

  • Site Contact Information

    Barrow Neurological Institute
    Gale Kittle / .(JavaScript must be enabled to view this email address) / Phoenix, Arizona 85013
    United States

    California Pacific Medical Center
    Marguerite Engel / .(JavaScript must be enabled to view this email address) / 415-309-5178
    San Diego, California 94114
    United States

    Cedars-Sinai Medical Center
    Ashley Fetterman / .(JavaScript must be enabled to view this email address) / Los Angeles, California 90048
    United States

    Stanford University
    Shirley Paulose / .(JavaScript must be enabled to view this email address) / 650-724-3792
    Stanford, California 94305
    United States

    Hospital for Special Care
    Boguslawa (Agnes) Koczon-Jaremko; Nicole Allard / .(JavaScript must be enabled to view this email address) / 860-612-6356; 860-612-6356
    New Britain, Connecticut 06053
    United States

    University of Florida, Jacksonville
    Lisa Smith / .(JavaScript must be enabled to view this email address) / Jacksonville, Florida 32209
    United States

    Mayo Clinic Florida
    Pamela DeSaro / .(JavaScript must be enabled to view this email address) / 904-953-6915
    Jacksonville, Florida 32224
    United States

    University of Iowa
    Jeri Sieren / .(JavaScript must be enabled to view this email address) / 319-356-8744
    Des Moines, Iowa 52242
    United States

    Massachusetts General Hospital
    Fernando Gonterman / .(JavaScript must be enabled to view this email address) / 617-726-0563
    Boston, Massachusetts 02114
    United States

    Henry Ford Health Systems
    Beverly Stallings / .(JavaScript must be enabled to view this email address) / 313-916-3955
    Detroit, Michigan 48202
    United States

    St. Louis University
    Craig Dedert / .(JavaScript must be enabled to view this email address) / 314-577-8461
    St. Louis, Missouri 63103
    United States

    Neurology Associates P.C.
    Rebecca Wills / .(JavaScript must be enabled to view this email address) / 402-413-3187
    Lincoln, Nebraska 68510
    United States

    Hospital for Special Surgery
    Lindsay Kaplan / .(JavaScript must be enabled to view this email address) / 646-797-8657
    New York, New York 10021
    United States

    SUNY Upstate Medical University
    Andrea McGlond / .(JavaScript must be enabled to view this email address) / 315-464-6409
    Syracuse, New York 13210
    United States

    Carolinas Health Care
    Cynthia Larry / .(JavaScript must be enabled to view this email address) / 704-446-1987
    Charlotte, North Carolina 28203
    United States

    Wake Forest University
    Mozhdeh Marandi / .(JavaScript must be enabled to view this email address) / 336-713-8577
    Winston-Salem, North Carolina 27106
    United States

    Cleveland Clinic
    Irys Caristo / .(JavaScript must be enabled to view this email address) / Cleveland, Ohio 44195
    United States

    Ohio State University
    Colleen Pineda / .(JavaScript must be enabled to view this email address) / 614-685-5661
    Columbus, Ohio 43210
    United States

    Providence ALS Center
    Chad Parks / .(JavaScript must be enabled to view this email address) / 503-962-1171
    Portland, Oregon 97213
    United States

    University of Pittsburgh
    Danielle Rowlands / .(JavaScript must be enabled to view this email address) / 412-648-9053
    Pittsburgh, Pennsylvania 15260
    United States

    Drexel University
    Christine Barr / .(JavaScript must be enabled to view this email address) / 267-507-2633
    Philadelphia, Pennsylvania 19104
    United States

    University of Texas
    Pamela Kittrell / .(JavaScript must be enabled to view this email address) / 210-450-0524
    San Antonio, Texas 78705
    United States

    University of Texas Southwestern
    Nina Gorham / .(JavaScript must be enabled to view this email address) / 214-648-9518
    Dallas, Texas 75390
    United States

    University of Vermont
    Shannon Lucy / .(JavaScript must be enabled to view this email address) / 802-656-4582
    Burlington, Vermont 05405
    United States

    Virginia Mason Medical Center
    Leslie Stevens / .(JavaScript must be enabled to view this email address) / 206-342-6929
    Seattle, Washington 98101
    United States