Amyotrophic Lateral Sclerosis (ALS)NCT01605006
Humanitarian Device Exemption Post-Approval Study of NeuRx Diaphragm Pacing System for Amyotrophic Lateral Sclerosis
A study testing NeuRx Diaphragm Pacing System (DPS) in people with Amyotrophic Lateral Sclerosis (ALS), run by Synapse Biomedical at 11 sites in United States.
Trial summary
This post-approval study will follow 60 participants who have ALS, documented chronic hypoventilation, and bilateral phrenic nerve function, and who undergo the surgical implantation procedure to receive the NeuRx Diaphragm Pacing System device. 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
- 97 participants
- Where
- 11 sitesUnited States
- Study dates
- Jul 2012to Feb 2017
- Phase
- Interventional
- 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 or older.
- Participants with familial or sporadic ALS diagnosed as laboratory-supported probable, probable, or definite according to the World Federation of Neurology El Escorial criteria.
- Bilateral phrenic nerve function clinically acceptable as demonstrated by bilateral diaphragm movement with fluoroscopic sniff test or with EMG recordings and nerve conduction times.
- Chronic hypoventilation was documented by at least one of the following:
- FVC less than 50% predicted, or
- |MIP| less than 60 cmH2O, or
Show all 11Show fewer
- PaCO2 greater than or equal to 45 mmHg, or
- Nocturnal SaO2 less than or equal to 88% for at least five continuous minutes
- Suitable surgical candidate.
- Negative pregnancy test in female participants of childbearing potential.
- Informed consent from patient or designated representative.
Who cannot join
- Underlying cardiac or pulmonary disease that would increase the risk of general anesthesia.
- Underlying pulmonary diseases that were present prior to ALS that would affect pulmonary tests independent of ALS.
- Uncontrolled excessive secretions.
- FVC less than 45% predicted at time of surgery.
- Preexisting implanted electrical device such as pacemaker or cardiac defibrillator.
- Pre-existing diaphragm abnormality such as a hiatal hernia or paraesophageal hernia of abdominal contents going into the thoracic cavity.
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
Northwestern University
Chicago, Illinois, 60611
University Hospitals Case Medical Center
Cleveland, Ohio, 44106
University of Colorado Denver
Denver, Colorado, 80045
Duke University
Durham, North Carolina, 27705
Mayo Clinic
Jacksonville, Florida, 32224
University of Kansas Medical Center
Kansas City, Kansas, 66160
Cedars-Sinai Medical Center
Los Angeles, California, 90048
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Providence St. Vincent Medical Center
Portland, Oregon, 97213
California Pacific Medical Center -- Forbes Norris MDA/ALS Research Center
San Francisco, California, 94115
Stony Brook University
Stony Brook, New York, 11794
About this study
Official title: HDE Post-Approval Study (PAS) of NeuRx DPS for ALS
This post-approval study will follow 60 participants who have ALS, documented chronic hypoventilation, and bilateral phrenic nerve function, and who undergo the surgical implantation procedure to receive the NeuRx Diaphragm Pacing System device. Participants who are successfully implanted with the device will use it for daily diaphragm conditioning sessions. Participants will be followed for at least two years (until the last enrolled participant reaches the 2-year follow-up visit). Safety and probable benefit outcome measures will be assessed.
Read the detailed description
This is a prospective, non-randomized, open-label, interventional, post-approval (FDA) study of the NeuRx Diaphragm Pacing System (DPS) device. The study will enroll 60 participants who have amyotrophic lateral sclerosis (ALS), meet the FDA-approved device indications for use, and undergo the surgical implantation procedure to receive the device. The device is intended for use in ALS patients with a stimulatable diaphragm (both right and left portions) as demonstrated by voluntary contraction or phrenic nerve conduction studies, and who are experiencing chronic hypoventilation (CH), but not progressed to an FVC less than 45% predicted. Participants who are successfully implanted with the device will use it for daily diaphragm conditioning sessions. Participants will be followed for at least two years (until the last enrolled participant reaches the 2-year follow-up visit). Safety and probable benefit outcome measures will be assessed. The primary objective of the study is: (1) (Safety) Characterize the types and frequency of major device-related adverse events (AEs) over the time of device use. Secondary objectives of the study are: (2) (Safety) Determine whether the frequency of major device-related AEs increases dramatically toward end of life; and (3) (Probable Benefit) Determine whether there is a relationship between survival time and onset type (bulbar and limb), time from onset to treatment, and use of NIV, riluzole, or PEG in patients treated with the device.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- NeuRx Diaphragm Pacing System (DPS)
- Keywords
- amyotrophic lateral sclerosis, ALS, motor neuron disease, diaphragm, diaphragm pacing, diaphragmatic pacing, phrenic pacing, phrenic nerve, phrenic nerve stimulation, NeuRx DPS, DPS, chronic hypoventilation, respiration, ventilation, breathing, Humanitarian Device Exemption, HDE, post-approval study
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Respiratory Aspiration
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
Also called diaphragm pacing, diaphragmatic pacing, phrenic nerve stimulation
The NeuRx DPS is a percutaneous, intramuscular, diaphragm stimulation system which is implanted using standard laparoscopic surgical techniques in an outpatient procedure. The implanted intramuscular diaphragm electrodes are connected to a four channel external stimulator at a percutaneous exit site. DPS is designed to help ALS patients breathe by providing conditioning stimulation of the diaphragm muscles. Recommended frequency of diaphragm conditioning is at least 3 times per day with each session lasting at least 30 minutes. Patients may find it helpful to use the DPS for longer periods to help with breathing. DPS may be used at the same time as non-invasive ventilation. Patents may also sleep with the DPS to assist with breathing difficulties at night.
| Study group | Role | What they receive |
|---|---|---|
| NeuRx Diaphragm Pacing System (DPS) | Other | Surgical implantation of the NeuRx DPS (on label use). Receives: Device: NeuRx Diaphragm Pacing System (DPS) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety Outcome Measure -- Occurrence of major device-related (including procedure-related) adverse events as defined below.
- Serious capnothorax requiring invasive intervention
- Mechanical ventilation for 24 hours or longer post-procedure
- Post-procedure extubation failure resulting in permanent tracheostomy ventilation
- Perioperative complication which delays initiation of NeuRx DPS therapy
- Severe discomfort due to stimulation which is unable to be tolerated or resolved
- Device malfunction which interrupts or causes an undesired diminution of NeuRx DPS therapy
- Electrode dislodgement from the diaphragm
- Wire infection
- Any other device- or procedure-related serious adverse event
Measured: follow-up assessments at 3-month intervals
Secondary outcomes (1)
Probable Benefit Outcome Measure
Survival, defined as time to (a) death or (b) permanent tracheostomy mechanical ventilation (PTV) with discontinuation of pacing. (All deaths and PTV events will be reported regardless of relationship to the device or procedure.)
Measured: follow-up assessments at 3-month intervals
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2012 (estimated)
- Study startJul 2012
- Primary completionFeb 2017
- Study completionFeb 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 97 participants (actual)
Sponsor and investigators
- Lead sponsor
- Synapse Biomedical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Robert G. Miller, M.D., Principal investigator, Forbes Norris MDA/ALS Research Center, California Pacific Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01605006
- Sponsor's protocol ID
- CLIN 20-0009-0020
- Status last verified
- Mar 2020
- First submitted
- 22 May 2012
- Last updated
- 27 Mar 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- HDE Post-Approval Study (PAS) of NeuRx DPS for ALS
- ClinicalTrials.gov ID
- NCT01605006
- Acronym / sponsor ID
- CLIN 20-0009-0020
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Keywords
- amyotrophic lateral sclerosis, ALS, motor neuron disease, diaphragm, diaphragm pacing, diaphragmatic pacing, phrenic pacing, phrenic nerve
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 97 participants (actual)
- Lead sponsor
- Synapse BiomedicalIndustry (pharma, biotech, devices)
- Study start
- Jul 2012 (actual)
- Primary completion
- Feb 2017 (actual)
- Study completion
- Feb 2017 (actual)
- First posted
- 24 May 2012 (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.
This post-approval study will follow 60 participants who have ALS, documented chronic hypoventilation, and bilateral phrenic nerve function, and who undergo the surgical implantation procedure to receive the NeuRx Diaphragm Pacing System device. Participants who are successfully implanted with the device will use it for daily diaphragm conditioning sessions. Participants will be followed for at least two years (until the last enrolled participant reaches the 2-year follow-up visit). Safety and probable benefit outcome measures will be assessed.
Detailed description
This is a prospective, non-randomized, open-label, interventional, post-approval (FDA) study of the NeuRx Diaphragm Pacing System (DPS) device. The study will enroll 60 participants who have amyotrophic lateral sclerosis (ALS), meet the FDA-approved device indications for use, and undergo the surgical implantation procedure to receive the device. The device is intended for use in ALS patients with a stimulatable diaphragm (both right and left portions) as demonstrated by voluntary contraction or phrenic nerve conduction studies, and who are experiencing chronic hypoventilation (CH), but not progressed to an FVC less than 45% predicted. Participants who are successfully implanted with the device will use it for daily diaphragm conditioning sessions. Participants will be followed for at least two years (until the last enrolled participant reaches the 2-year follow-up visit). Safety and probable benefit outcome measures will be assessed. The primary objective of the study is: (1) (Safety) Characterize the types and frequency of major device-related adverse events (AEs) over the time of device use. Secondary objectives of the study are: (2) (Safety) Determine whether the frequency of major device-related AEs increases dramatically toward end of life; and (3) (Probable Benefit) Determine whether there is a relationship between survival time and onset type (bulbar and limb), time from onset to treatment, and use of NIV, riluzole, or PEG in patients treated with the device.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NeuRx Diaphragm Pacing System (DPS) | Other | Device: NeuRx Diaphragm Pacing System (DPS)Surgical implantation of the NeuRx DPS (on label use). |
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 or older.
- Participants with familial or sporadic ALS diagnosed as laboratory-supported probable, probable, or definite according to the World Federation of Neurology El Escorial criteria.
- Bilateral phrenic nerve function clinically acceptable as demonstrated by bilateral diaphragm movement with fluoroscopic sniff test or with EMG recordings and nerve conduction times.
- Chronic hypoventilation was documented by at least one of the following:
- Suitable surgical candidate.
Key exclusion criteria (6 listed)
- Underlying cardiac or pulmonary disease that would increase the risk of general anesthesia.
- Underlying pulmonary diseases that were present prior to ALS that would affect pulmonary tests independent of ALS.
- Uncontrolled excessive secretions.
- FVC less than 45% predicted at time of surgery.
- Preexisting implanted electrical device such as pacemaker or cardiac defibrillator.
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)Also: diaphragm pacing, diaphragmatic pacing, phrenic nerve stimulationThe NeuRx DPS is a percutaneous, intramuscular, diaphragm stimulation system which is implanted using standard laparoscopic surgical techniques in an outpatient procedure. The implanted intramuscular diaphragm electrodes are connected to a four channel external stimulator at a percutaneous exit site. DPS is designed to help ALS patients breathe by providing conditioning stimulation of the diaphragm muscles. Recommended frequency of diaphragm conditioning is at least 3 times per day with each session lasting at least 30 minutes. Patients may find it helpful to use the DPS for longer periods to help with breathing. DPS may be used at the same time as non-invasive ventilation. Patents may also sleep with the DPS to assist with breathing difficulties at night. | Device | NeuRx Diaphragm Pacing System (DPS) |
Primary outcome
Safety Outcome Measure -- Occurrence of major device-related (including procedure-related) adverse events as defined below.
Time frame: follow-up assessments at 3-month intervals
* Serious capnothorax requiring invasive intervention * Mechanical ventilation for 24 hours or longer post-procedure * Post-procedure extubation failure resulting in permanent tracheostomy ventilation * Perioperative complication which delays initiation of NeuRx DPS therapy * Severe discomfort due to stimulation which is unable to be tolerated or resolved * Device malfunction which interrupts or causes an undesired diminution of NeuRx DPS therapy * Electrode dislodgement from the diaphragm * Wire infection * Any other device- or procedure-related serious adverse event
1 secondary outcome
Probable Benefit Outcome Measure
Time frame: follow-up assessments at 3-month intervals
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 27 Mar 2020.
- FDA Summary of Safety and Probable Benefit for Humanitarian Device Exemption (Approved September 28, 2011)Link provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Robert G. Miller, M.D. principal investigator
Forbes Norris MDA/ALS Research Center, California Pacific Medical Center
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing NeuRx Diaphragm Pacing System (DPS) in people with Amyotrophic Lateral Sclerosis (ALS), run by Synapse Biomedical at 11 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 6 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 27 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 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.
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?
Safety Outcome Measure -- Occurrence of major device-related (including procedure-related) adverse events as defined below. (time frame: follow-up assessments at 3-month intervals). 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 27 Mar 2020.