Trial Outcomes & Findings for Non-invasive Vagus Nerve Stimulation (nVNS) in Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) (NCT NCT03772717)
NCT ID: NCT03772717
Last Updated: 2023-07-14
Results Overview
Motor nerve conduction studies are used to examine conduction of electrical impulses along nerves. Electrodes are placed on the skin in specific areas to evaluate peripheral nerves. An electrode stimulates a nerve while the receiving site records how well electrical impulses are being conducted along the nerve. Latency is the time it takes in milliseconds (ms) for the electrical impulse to travel to the site receiving the stimulation.
TERMINATED
NA
2 participants
Baseline, Month 12, Month 24
2023-07-14
Participant Flow
Participants were recruited from Children's Healthcare of Atlanta in Atlanta, Georgia, USA. Participant enrollment began February 22, 2022 and the study was terminated June 30, 2022, prior to any participant reaching the first follow-up visit at Month 6.
Participant milestones
| Measure |
Non-invasive Vagus Nerve Stimulation (nVNS)
Participants chronic inflammatory demyelinating polyneuropathy (CIDP) used the electrical neuromuscular stimulator device, VitalStim 400, while continuing to take their standard of care medication.
Non-invasive vagus nerve stimulation (nVNS): The nVNS study intervention is delivered using a handheld electrical neuromuscular stimulator device (VitalStim 400). Participants are instructed to deliver nVNS twice per day for 60 minutes each time at least 5 days per week. The two electrodes for the device are placed on the subjects left cervical (neck) region. Parents are trained on where to place electrodes, how to ensure that the electrodes make a good contact with the skin, and how to set the stimulation parameters. The stimulation frequency (number of pulses) and amplitude (amount of current) are set during the initial baseline session in the clinic at a level that prevents discomfort and does not impact cardiorespiratory parameters. The stimulator is placed in a comfortable position, such as next to the pillow. The stimulators are battery-powered and allow configuration of the stimulation parameters to the comfort of the patient.
Standard of care treatment: Patients are asked to continue their standard medication regimens which include in most cases will involve 3 weekly infusions of intravenous immunoglobulin (IVIG) (1 gm/kg) and rarely plasma exchange (PLEX).
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|---|---|
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Overall Study
STARTED
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2
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Overall Study
COMPLETED
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0
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Overall Study
NOT COMPLETED
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2
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Reasons for withdrawal
| Measure |
Non-invasive Vagus Nerve Stimulation (nVNS)
Participants chronic inflammatory demyelinating polyneuropathy (CIDP) used the electrical neuromuscular stimulator device, VitalStim 400, while continuing to take their standard of care medication.
Non-invasive vagus nerve stimulation (nVNS): The nVNS study intervention is delivered using a handheld electrical neuromuscular stimulator device (VitalStim 400). Participants are instructed to deliver nVNS twice per day for 60 minutes each time at least 5 days per week. The two electrodes for the device are placed on the subjects left cervical (neck) region. Parents are trained on where to place electrodes, how to ensure that the electrodes make a good contact with the skin, and how to set the stimulation parameters. The stimulation frequency (number of pulses) and amplitude (amount of current) are set during the initial baseline session in the clinic at a level that prevents discomfort and does not impact cardiorespiratory parameters. The stimulator is placed in a comfortable position, such as next to the pillow. The stimulators are battery-powered and allow configuration of the stimulation parameters to the comfort of the patient.
Standard of care treatment: Patients are asked to continue their standard medication regimens which include in most cases will involve 3 weekly infusions of intravenous immunoglobulin (IVIG) (1 gm/kg) and rarely plasma exchange (PLEX).
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|---|---|
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Overall Study
Withdrawal by Subject
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2
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Baseline Characteristics
Non-invasive Vagus Nerve Stimulation (nVNS) in Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Baseline characteristics by cohort
| Measure |
Non-invasive Vagus Nerve Stimulation (nVNS)
n=2 Participants
Participants with CIDP used the electrical neuromuscular stimulator device, VitalStim 400, while continuing to take their standard of care medication.
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|---|---|
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Age, Categorical
<=18 years
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2 Participants
n=5 Participants
|
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Age, Categorical
Between 18 and 65 years
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0 Participants
n=5 Participants
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Age, Categorical
>=65 years
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0 Participants
n=5 Participants
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Sex: Female, Male
Female
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1 Participants
n=5 Participants
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Sex: Female, Male
Male
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1 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=5 Participants
|
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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2 Participants
n=5 Participants
|
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Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=5 Participants
|
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
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|
Race (NIH/OMB)
Asian
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
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0 Participants
n=5 Participants
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Race (NIH/OMB)
White
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1 Participants
n=5 Participants
|
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Race (NIH/OMB)
More than one race
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1 Participants
n=5 Participants
|
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Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
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Region of Enrollment
United States
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2 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: Baseline, Month 12, Month 24Population: Participants withdrew from the study prior to completing any follow-up study visits.
Motor nerve conduction studies are used to examine conduction of electrical impulses along nerves. Electrodes are placed on the skin in specific areas to evaluate peripheral nerves. An electrode stimulates a nerve while the receiving site records how well electrical impulses are being conducted along the nerve. Latency is the time it takes in milliseconds (ms) for the electrical impulse to travel to the site receiving the stimulation.
Outcome measures
| Measure |
Non-invasive Vagus Nerve Stimulation (nVNS)
n=2 Participants
Participants with CIDP used the electrical neuromuscular stimulator device, VitalStim 400, while continuing to take their standard of care medication.
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|---|---|
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Nerve Conduction Study - Distal Latency
Baseline
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4.8 ms
Standard Deviation 0.28
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PRIMARY outcome
Timeframe: Baseline, Month 12, Month 24Population: F wave latency was not performed for one participant at the baseline visit. Participants withdrew from the study prior to completing any follow-up study visits.
Motor nerve conduction studies are used to examine conduction of electrical impulses along nerves. Electrodes are placed on the skin in specific areas to evaluate peripheral nerves. An electrode stimulates a nerve while the receiving site records how well electrical impulses are being conducted along the nerve. F wave latency is the time it takes in milliseconds (ms) for an electrical signal to travel from the stimulating electrode to the distal muscle and back to the stimulating site. F waves are used to assess polyneuropathy and F wave latency can be extended or even absent in persons with CIDP.
Outcome measures
| Measure |
Non-invasive Vagus Nerve Stimulation (nVNS)
n=1 Participants
Participants with CIDP used the electrical neuromuscular stimulator device, VitalStim 400, while continuing to take their standard of care medication.
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|---|---|
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Nerve Conduction Study - F Wave Latency
Baseline
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20.12 ms
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PRIMARY outcome
Timeframe: Baseline, Month 12, Month 24Population: Participants withdrew from the study prior to completing any follow-up study visits.
Motor nerve conduction studies are used to examine conduction of electrical impulses along nerves. Electrodes are placed on the skin in specific areas to evaluate peripheral nerves. An electrode stimulates a nerve while the receiving site records how well electrical impulses are being conducted along the nerve. Conduction velocity measures the rate of impulse conduction in meters per second (m/s) and is often decreased in patients with CIDP as myelination is affected.
Outcome measures
| Measure |
Non-invasive Vagus Nerve Stimulation (nVNS)
n=2 Participants
Participants with CIDP used the electrical neuromuscular stimulator device, VitalStim 400, while continuing to take their standard of care medication.
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|---|---|
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Nerve Conduction Study - Conduction Velocity
Baseline
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37.5 m/s
Standard Deviation 10.61
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PRIMARY outcome
Timeframe: Baseline, Month 12, Month 24Population: Participants withdrew from the study prior to completing any follow-up study visits.
Motor nerve conduction studies are used to examine conduction of electrical impulses along nerves. Electrodes are placed on the skin in specific areas to evaluate peripheral nerves. An electrode stimulates a nerve while the receiving site records how well electrical impulses are being conducted along the nerve. Conduction amplitude is the size of the response to electrical stimulation, measured in millivolts (mV). Reduced amplitude indicates axon loss.
Outcome measures
| Measure |
Non-invasive Vagus Nerve Stimulation (nVNS)
n=2 Participants
Participants with CIDP used the electrical neuromuscular stimulator device, VitalStim 400, while continuing to take their standard of care medication.
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|---|---|
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Nerve Conduction Study - Conduction Amplitude
Baseline
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4.15 mV
Standard Deviation 1.20
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PRIMARY outcome
Timeframe: Baseline, Month 6, Month 12, Month 18, Month 24Population: Participants withdrew from the study prior to completing any follow-up study visits.
Hand grip strength is assessed with a Jamar Handheld Dynamometer for children ages 5-18 years and measures strength in kilograms (kg). Both right and left hand grip strength were measured and the best of three attempts were used for each hand. Increased hand strength is an indicator of effective treatment.
Outcome measures
| Measure |
Non-invasive Vagus Nerve Stimulation (nVNS)
n=2 Participants
Participants with CIDP used the electrical neuromuscular stimulator device, VitalStim 400, while continuing to take their standard of care medication.
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|---|---|
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Hand Grip Strength
Baseline - Right Hand
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19.1 kg
Standard Deviation 2.97
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Hand Grip Strength
Baseline - Left Hand
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18.55 kg
Standard Deviation 2.62
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PRIMARY outcome
Timeframe: Baseline, Month 6, Month 12, Month 18, Month 24Population: Participants withdrew from the study prior to completing any follow-up study visits.
The Rasch-built Overall Disability Scale (R-ODS) used for those with Guillain-Barré syndrome (GBS), chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), and gammopathy-related polyneuropathy (MGUSP) is a 24-item scale asking respondents to rate how greatly polyneuropathy impacts activities. Responses are given on a scale of 0 to 2 where 0 indicates it is not possible for the respondent to perform the task and 2 means that the task can be performed without difficulty. Total scores range from 0 to 48 and higher scores indicate greater ability to perform daily and social tasks.
Outcome measures
| Measure |
Non-invasive Vagus Nerve Stimulation (nVNS)
n=2 Participants
Participants with CIDP used the electrical neuromuscular stimulator device, VitalStim 400, while continuing to take their standard of care medication.
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|---|---|
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Rasch-built Overall Disability Scale (R-ODS) for CIDP Score
Baseline
|
45.5 score on a scale
Standard Deviation 2.12
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SECONDARY outcome
Timeframe: Baseline, Month 6, Month 12, Month 18, Month 24Population: One participant had TNF-α value at baseline that was below the limit of detection (\<1.7 pg/mL) so the observation for this participant is not included as a summary score cannot be calculated. Participants withdrew from the study prior to completing any follow-up study visits.
The impact of treatment on serum cytokine profiles will be assessed by measuring TNF-α. Serum cytokine levels will be statistically analyzed on a per patient basis, with each patient's baseline measurements used for comparison. TNF-α is elevated in CIDP patients and a decrease in serum TNF-α is an indication of effective treatment.
Outcome measures
| Measure |
Non-invasive Vagus Nerve Stimulation (nVNS)
n=1 Participants
Participants with CIDP used the electrical neuromuscular stimulator device, VitalStim 400, while continuing to take their standard of care medication.
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|---|---|
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Tumor Necrosis Factor (TNF)-α
Baseline
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2.9 picograms per milliliter (pg/mL)
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SECONDARY outcome
Timeframe: Baseline, Month 6, Month 12, Month 18, Month 24Population: One participant had a IL-1β value at baseline that was below the limit of detection (\<6.5 pg/mL) so the observation for this participant is not included as a summary score cannot be calculated. Participants withdrew from the study prior to completing any follow-up study visits.
The impact of treatment on serum cytokine profiles will be assessed by measuring IL-1β. Serum cytokine levels will be statistically analyzed on a per patient basis, with each patient's baseline measurements used for comparison. IL-1β is elevated in CIDP patients and a decrease in IL-1β values is an indication of effective treatment.
Outcome measures
| Measure |
Non-invasive Vagus Nerve Stimulation (nVNS)
n=1 Participants
Participants with CIDP used the electrical neuromuscular stimulator device, VitalStim 400, while continuing to take their standard of care medication.
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|---|---|
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Interleukin (IL)-1β
Baseline
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13.4 pg/mL
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Adverse Events
Non-invasive Vagus Nerve Stimulation (nVNS)
Serious adverse events
| Measure |
Non-invasive Vagus Nerve Stimulation (nVNS)
n=2 participants at risk
Participants with CIDP used the electrical neuromuscular stimulator device, VitalStim 400, while continuing to take their standard of care medication.
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|---|---|
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Nervous system disorders
Tingling, numbness, and pain in limbs
|
50.0%
1/2 • Information on adverse events was collected from the time individuals gave consent to participate in the study until the participant withdrew from the study (up to 3 months).
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Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place