Amyotrophic Lateral Sclerosis (ALS)Phase 2DPS in ALSNCT01938495
Diaphragm Pacing System (DPS) In Participants With Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing NeuRx® Diaphragm Pacing System™ (DPS) in people with Amyotrophic Lateral Sclerosis (ALS), run by Barrow Neurological Institute at 25 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 21 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- NeuRx® Diaphragm Pacing System™ (DPS)
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 52 participants
- Where
- 25 sitesUnited States
- Study dates
- Aug 2013to Apr 2017
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
25 sites in 1 country.
25 sites in 1 country
United States25 sites
Massachusetts General Hospital
Boston, Massachusetts
University of Vermont
Burlington, Vermont
Carolinas Health Care
Charlotte, North Carolina
Cleveland Clinic
Cleveland, Ohio
Ohio State University
Columbus, Ohio
University of Texas Southwestern
Dallas, Texas
Henry Ford Health Systems
Detroit, Michigan
University of Iowa
Iowa City, Iowa
Mayo Clinic Florida
Jacksonville, Florida
University of Florida, Jacksonville
Jacksonville, Florida
Neurology Associates P.C.
Lincoln, Nebraska
Cedars-Sinai Medical Center
Los Angeles, California
Hospital for Special Care
New Britain, Connecticut
Hospital for Special Surgery
New York, New York
Drexel University
Philadelphia, Pennsylvania
Barrow Neurological Institute
Phoenix, Arizona
University of Pittsburgh
Pittsburgh, Pennsylvania
Providence ALS Center
Portland, Oregon
University of Texas
San Antonio, Texas
California Pacific Medical Center
San Diego, California
Virginia Mason Medical Center
Seattle, Washington
St Louis University
St Louis, Missouri
Stanford University
Stanford, California
SUNY Upstate Medical University
Syracuse, New York
Wake Forest University
Winston-Salem, North Carolina
About this study
Official title: Multi-Center, Randomized Controlled Study of the NeuRx® Diaphragm Pacing System™ (DPS)In Participants With Amyotrophic Lateral Sclerosis (ALS)
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.
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- NeuRx® Diaphragm Pacing System™ (DPS)
- Keywords
- ALS, DPS, Diaphragm Pacing
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Survival
Measured: Subjects will be assessed on a monthly basis for an 18 month period.
Secondary outcomes (1)
quality of life
Measured: Subjects will be assessed on a monthly basis for an 18 month period.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2013 (estimated)
- Study startAug 2013
- Primary completionJan 2017
- Study completionApr 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Barrow Neurological Institute
- Sponsor type
- University, hospital or other
- Collaborators
- ALS Association, Muscular Dystrophy Association, Synapse Biomedical
- Responsible party
- Principal investigator, Jeremy Shefner, Barrow Neurological Institute
- Investigators
- Jeremy Shefner, MD, PhD, Principal investigator, Barrow Neurological InstituteJonathan Katz, MD, Principal investigator, California Pacific Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01938495
- Sponsor's protocol ID
- 2013P001504
- Acronym
- DPS in ALS
- Status last verified
- Mar 2023
- First submitted
- 23 Aug 2013
- Last updated
- 9 Mar 2023
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-Center, Randomized Controlled Study of the NeuRx® Diaphragm Pacing System™ (DPS)In Participants With Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT01938495
- Acronym / sponsor ID
- DPS in ALS / 2013P001504
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Keywords
- ALS, DPS, Diaphragm Pacing
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Barrow Neurological InstituteUniversity, hospital or other
- Collaborators
- ALS Association, Muscular Dystrophy Association, Synapse Biomedical
- Study start
- Aug 2013
- Primary completion
- Jan 2017 (actual)
- Study completion
- Apr 2017 (actual)
- First posted
- 10 Sep 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NeuRx® Diaphragm Pacing System™ (DPS) | Experimental | Device: 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. |
| 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. |
Who may qualify
- Ages
- 21 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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:
- 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).
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Device | NeuRx® Diaphragm Pacing System™ (DPS) |
Primary outcome
Survival
Time frame: Subjects will be assessed on a monthly basis for an 18 month period.
1 secondary outcome
quality of life
Time frame: Subjects will be assessed on a monthly basis for an 18 month period.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2023.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jeremy Shefner, MD, PhD principal investigator
Barrow Neurological Institute
Jonathan Katz, MD principal investigator
California Pacific Medical Center
25 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NeuRx® Diaphragm Pacing System™ (DPS) in people with Amyotrophic Lateral Sclerosis (ALS), run by Barrow Neurological Institute at 25 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Survival (time frame: Subjects will be assessed on a monthly basis for an 18 month period.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Mar 2023.