NeuRx Diaphram Pacing System (DPS) Use in Amyotrophic Lateral Sclerosis (ALS) (PARADIGM)
Primary Purpose
Amyotrophic Lateral Sclerosis
Status
Withdrawn
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
NeuRx DPS
Sponsored by
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring Diaphragm Pacing
Eligibility Criteria
Inclusion Criteria:
- Age 21 or older
- Familial or sporadic ALS diagnosed as possible, 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 phrenic nerve conduction studies
Chronic hypoventilation was documented by at least one of the following:
- MIP (respiratory muscle strength) less than 60 cmH2O, or
- Forced Vital Capacity (FVC) less than 50% predicted, or
- PaCO2 greater than or equal to 45 mmHg, or
- Nocturnal SaO2 (arterial oxygen saturation) less than or equal to 88% for at least five continuous minutes
- Initiation of non-invasive ventilation at or before time of enrollment
- Suitable surgical candidate to receive diaphragm pacing stimulation
- Negative pregnancy test in female participants of childbearing potential (treatment group)
- Informed consent from patient or designated representative
Exclusion Criteria:
- FVC less than 45% predicted within 10 days prior to surgery.
- Participation in simultaneous therapeutic clinical trial which could affect the safety or benefit outcomes of this study.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
No Intervention
Arm Label
NeuRx DPS
No DPS
Arm Description
Subjects who have initiated noninvasive ventilation (NIV) and receive the NeuRx diaphragm pacing system (DPS).
Subjects who have initiated noninvasive ventilation (NIV) and do not receive the DPS device.
Outcomes
Primary Outcome Measures
Survival
Compare the survival of patients who initiated noninvasive ventilation (NIV).and who receive DPS to controls without DPS: (1) concurrent controls; (2) historical controls (e.g., Lechtzin and PRO-ACT databases); and (3) virtual controls (Origent).
Secondary Outcome Measures
Relationship between survival time and clinical features of ALS
Determine whether there is a relationship between survival time and ALS onset type.
Relationship between survival time and ALS assessment scores
Determine whether there is a relationship between survival time and Upper Motor Neuron (UMN)/ Lower Motor Neuron (LMN) assessment scores.
Relationship between survival time and onset of weakness from ALS to treatment
Relationship between survival time and ALS treatment interventions
Relationship between survival time and intraoperative strength of contraction
Characterize change in overall and respiratory function
Characterize longitudinal change in overall function (ALSFRS-R) before and after treatment with DPS compared to controls.
Characterize change in respiratory function
Characterize longitudinal change in respiratory function (FVC, MIP) before and after treatment with DPS compared to controls.
Types device and procedure-related adverse events
Frequency of device and procedure-related adverse events
Types of respiratory serious adverse events
Frequency of respiratory adverse events
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03034317
Brief Title
NeuRx Diaphram Pacing System (DPS) Use in Amyotrophic Lateral Sclerosis (ALS)
Acronym
PARADIGM
Official Title
Multi-Center, Concurrent-Controlled, On-Label Study of the NeuRx Diaphragm Pacing System (DPS) in Participants With Amyotrophic Lateral Sclerosis (ALS)
Study Type
Interventional
2. Study Status
Record Verification Date
March 2020
Overall Recruitment Status
Withdrawn
Why Stopped
No participants enrolled
Study Start Date
February 2, 2017 (Actual)
Primary Completion Date
February 8, 2018 (Actual)
Study Completion Date
February 12, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Synapse Biomedical
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
Yes
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this research study is to collect more information about the use, safety, and effectiveness of the NeuRx DPS® in ALS patients.
Detailed Description
Characterize the safety and benefit of on-label Humanitarian Device Exemption (HDE) use of DPS in patients with ALS who initiated noninvasive ventilation (NIV).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
Diaphragm Pacing
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Masking Description
Open label
Allocation
Non-Randomized
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
NeuRx DPS
Arm Type
Active Comparator
Arm Description
Subjects who have initiated noninvasive ventilation (NIV) and receive the NeuRx diaphragm pacing system (DPS).
Arm Title
No DPS
Arm Type
No Intervention
Arm Description
Subjects who have initiated noninvasive ventilation (NIV) and do not receive the DPS device.
Intervention Type
Device
Intervention Name(s)
NeuRx DPS
Other Intervention Name(s)
Diaphragm Pacing System
Intervention Description
Patients who are diagnosed with ALS, have initiated NIV and choose to have the NeuRx DPS implanted.
Primary Outcome Measure Information:
Title
Survival
Description
Compare the survival of patients who initiated noninvasive ventilation (NIV).and who receive DPS to controls without DPS: (1) concurrent controls; (2) historical controls (e.g., Lechtzin and PRO-ACT databases); and (3) virtual controls (Origent).
Time Frame
24 months
Secondary Outcome Measure Information:
Title
Relationship between survival time and clinical features of ALS
Description
Determine whether there is a relationship between survival time and ALS onset type.
Time Frame
24 months
Title
Relationship between survival time and ALS assessment scores
Description
Determine whether there is a relationship between survival time and Upper Motor Neuron (UMN)/ Lower Motor Neuron (LMN) assessment scores.
Time Frame
24 months
Title
Relationship between survival time and onset of weakness from ALS to treatment
Time Frame
24 months
Title
Relationship between survival time and ALS treatment interventions
Time Frame
24 months
Title
Relationship between survival time and intraoperative strength of contraction
Time Frame
24 months
Title
Characterize change in overall and respiratory function
Description
Characterize longitudinal change in overall function (ALSFRS-R) before and after treatment with DPS compared to controls.
Time Frame
24 months
Title
Characterize change in respiratory function
Description
Characterize longitudinal change in respiratory function (FVC, MIP) before and after treatment with DPS compared to controls.
Time Frame
24 months
Title
Types device and procedure-related adverse events
Time Frame
24 months
Title
Frequency of device and procedure-related adverse events
Time Frame
24 months
Title
Types of respiratory serious adverse events
Time Frame
24 months
Title
Frequency of respiratory adverse events
Time Frame
24 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Age 21 or older
Familial or sporadic ALS diagnosed as possible, 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 phrenic nerve conduction studies
Chronic hypoventilation was documented by at least one of the following:
MIP (respiratory muscle strength) less than 60 cmH2O, or
Forced Vital Capacity (FVC) less than 50% predicted, or
PaCO2 greater than or equal to 45 mmHg, or
Nocturnal SaO2 (arterial oxygen saturation) less than or equal to 88% for at least five continuous minutes
Initiation of non-invasive ventilation at or before time of enrollment
Suitable surgical candidate to receive diaphragm pacing stimulation
Negative pregnancy test in female participants of childbearing potential (treatment group)
Informed consent from patient or designated representative
Exclusion Criteria:
FVC less than 45% predicted within 10 days prior to surgery.
Participation in simultaneous therapeutic clinical trial which could affect the safety or benefit outcomes of this study.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Robert Miller, MD
Organizational Affiliation
California Pacific Medical Center
Official's Role
Principal Investigator
12. IPD Sharing Statement
Plan to Share IPD
No
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NeuRx Diaphram Pacing System (DPS) Use in Amyotrophic Lateral Sclerosis (ALS)
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