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

NCT ID: NCT01938495

Last Updated: 2023-03-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

52 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-08-31

Study Completion Date

2017-04-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Amyotrophic Lateral Sclerosis (ALS)

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

SUPPORTIVE_CARE

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

NeuRx® Diaphragm Pacing System™ (DPS)

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.

Group Type EXPERIMENTAL

NeuRx® Diaphragm Pacing System™ (DPS)

Intervention Type DEVICE

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.

Standard of Care

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

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

NeuRx® Diaphragm Pacing System™ (DPS)

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.

Intervention Type DEVICE

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.
Minimum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

ALS Association

OTHER

Sponsor Role collaborator

Muscular Dystrophy Association

OTHER

Sponsor Role collaborator

Synapse Biomedical

INDUSTRY

Sponsor Role collaborator

Barrow Neurological Institute

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Jeremy Shefner

MD, PhD

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Jeremy Shefner, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Barrow Neurological Institute

Jonathan Katz, MD

Role: PRINCIPAL_INVESTIGATOR

California Pacific Medical Center

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Henry Ford Health Systems

Detroit, Michigan, United States

Site Status

St Louis University

St Louis, Missouri, United States

Site Status

Neurology Associates P.C.

Lincoln, Nebraska, United States

Site Status

Hospital for Special Surgery

New York, New York, United States

Site Status

SUNY Upstate Medical University

Syracuse, New York, United States

Site Status

Carolinas Health Care

Charlotte, North Carolina, United States

Site Status

Barrow Neurological Institute

Phoenix, Arizona, United States

Site Status

Cedars-Sinai Medical Center

Los Angeles, California, United States

Site Status

California Pacific Medical Center

San Diego, California, United States

Site Status

Stanford University

Stanford, California, United States

Site Status

Hospital for Special Care

New Britain, Connecticut, United States

Site Status

Mayo Clinic Florida

Jacksonville, Florida, United States

Site Status

University of Florida, Jacksonville

Jacksonville, Florida, United States

Site Status

University of Iowa

Iowa City, Iowa, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Wake Forest University

Winston-Salem, North Carolina, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

Ohio State University

Columbus, Ohio, United States

Site Status

Providence ALS Center

Portland, Oregon, United States

Site Status

Drexel University

Philadelphia, Pennsylvania, United States

Site Status

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

University of Texas Southwestern

Dallas, Texas, United States

Site Status

University of Texas

San Antonio, Texas, United States

Site Status

University of Vermont

Burlington, Vermont, United States

Site Status

Virginia Mason Medical Center

Seattle, Washington, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Related Links

Access external resources that provide additional context or updates about the study.

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2013P001504

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

A Trial of Tocilizumab in ALS Subjects
NCT02469896 COMPLETED PHASE2
CNS10-NPC-GDNF for the Treatment of ALS
NCT02943850 COMPLETED PHASE1