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

NCT01938495 | Completed Phase 2 DMC

• 1 other identifier: 2013P001504
• Study Type: interventional
• Target: 52
• Locations: 1 country
• Sites: 25

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.

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Keywords

ALS; DPS; Diaphragm Pacing

Outcome Measures

Primary Outcomes (1)

• Survival

  Subjects will be assessed on a monthly basis for an 18 month period.

Secondary Outcomes (1)

• quality of life

  Subjects will be assessed on a monthly basis for an 18 month period.

Study Arms (2)

NeuRx® Diaphragm Pacing System™ (DPS)

EXPERIMENTAL

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.

Device: NeuRx® Diaphragm Pacing System™ (DPS)

Standard of Care

NO INTERVENTION

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

Interventions

NeuRx® Diaphragm Pacing System™ (DPS) 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.

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 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Barrow Neurological Institute: lead
• ALS Association: collaborator
• Muscular Dystrophy Association: collaborator
• Synapse Biomedical: collaborator

Study Sites (25)

Barrow Neurological Institute

Phoenix, Arizona, United States

Location

Cedars-Sinai Medical Center

Los Angeles, California, United States

Location

California Pacific Medical Center

San Diego, California, United States

Location

Stanford University

Stanford, California, United States

Location

Hospital for Special Care

New Britain, Connecticut, United States

Location

Mayo Clinic Florida

Jacksonville, Florida, United States

Location

University of Florida, Jacksonville

Jacksonville, Florida, United States

Location

University of Iowa

Iowa City, Iowa, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, United States

Location

Henry Ford Health Systems

Detroit, Michigan, United States

Location

St Louis University

St Louis, Missouri, United States

Location

Neurology Associates P.C.

Lincoln, Nebraska, United States

Location

Hospital for Special Surgery

New York, New York, United States

Location

SUNY Upstate Medical University

Syracuse, New York, United States

Location

Carolinas Health Care

Charlotte, North Carolina, United States

Location

Wake Forest University

Winston-Salem, North Carolina, United States

Location

Cleveland Clinic

Cleveland, Ohio, United States

Location

Ohio State University

Columbus, Ohio, United States

Location

Providence ALS Center

Portland, Oregon, United States

Location

Drexel University

Philadelphia, Pennsylvania, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Location

University of Texas Southwestern

Dallas, Texas, United States

Location

University of Texas

San Antonio, Texas, United States

Location

University of Vermont

Burlington, Vermont, United States

Location

Virginia Mason Medical Center

Seattle, Washington, United States

Location

Related Links

• Related Info

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Jeremy Shefner, MD, PhD

  Barrow Neurological Institute

  PRINCIPAL INVESTIGATOR

• Jonathan Katz, MD

  California Pacific Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD

Study Record Dates

• First Submitted: August 23, 2013
• First Posted: September 10, 2013
• Study Start: August 1, 2013
• Primary Completion: January 1, 2017
• Study Completion: April 1, 2017
• Last Updated: March 9, 2023

Record last verified: 2023-03

Locations

US (25)