About this trial
Systemic lupus erythematosus (SLE) is a chronic autoimmune inflammatory disease. Joint and muscle pain and fatigue are extremely common among patients and contribute to a reduced quality of life. Available therapies may be associated with significant side effects and many patients do not achieve an adequate response to these treatments. Therefore, there is an unmet need to develop new strategies to reduce pain and fatigue. Filling this need would significantly improve patients' quality of life. This trial will evaluate the effects of a novel approach, stimulating the vagus nerve, a nerve originating in the brain as a potential therapeutic intervention for treatment of musculoskeletal pain and fatigue. Vagus nerve stimulation has multiple beneficial effects and is one of the body's own ways to modulate the immune system. One can stimulate the vagus nerve via the skin at the neck or at specific locations in the ear, (transcutaneous vagus nerve stimulation: tcVNS). We recently completed a short, small scale randomized, placebo controlled trial of tcVNS in patients with SLE and observed dramatic benefits on musculoskeletal pain and fatigue. The treatment was safe without side effects. We are therefore proposing a longer trial to validate our initial findings and to look at durability. In this study, 18 patients with musculoskeletal pain will be followed for 2 months and will receive tcVNS or placebo (sham stimulation) for 5 minutes/day for 28 days. Patients will have a 1 out of 3 chance of receiving sham stimulation and neither the patient nor the evaluating investigator will know the actual treatment. The stimulations are self-administered, are non-painful and have not been associated with serious risks. After 28 days of stimulation, treatment will be discontinued and patients will be monitored for an additional 28 days to evaluate durability. Pain, fatigue and disease activity will be evaluated as well as possible side effects will be monitored throughout the trial. This study will also explore biologic mechanisms that may be responsible for the potential clinical effects. This will include possible effects of stimulation on gut permeability and the stool microbiome, areas that may play a significant role contributing to SLE disease and its manifestations. The development of an effective treatment without significant side effects would be extremely valuable and a significant advance for patients with SLE. If efficacious, tcVNS offers a non-toxic, non-immunosuppressive strategy to control two of the most common symptoms of this disease.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
SLE (defined by the ACR or SLICC criteria),
Musculoskeletal pain ≥ 4 on a non-anchored VAS 10 cm scale,
BILAG C or greater on the Musculoskeletal Domain of the BILAG 2004,
If on corticosteroids, the dose must be stable and ≤ 10 mg/day (prednisone or equivalent) for at least 14 days before baseline,
Disqualifiers
Initiation of immunosuppressive or antimalarial treatment within 3 months of baseline,
Treatment with cyclophosphamide within 2 months of baseline,
Initiation of anifrolumab within 3 months of baseline
Initiation of belimumab within 6 months of baseline,
Trial design
Parallel
Treatments tested in this trial
Active vagus nerve stimulation
DevicePatients will receive transcutaneous stimulation of the vagus nerve for 5 minutes daily for 28 consecutive days. The device is a handheld electrical pulse generator and a pair of electrodes will be placed on the skin for stimulation. The electrical pulse generator is turned on and the amplitude of stimulation increased to the greatest amount tolerated. Patients will be followed through day 57 to assess durability of the intervention.
Sham vagus nerve stimulation
DevicePatients will receive transcutaneous stimulation of the vagus nerve for 5 minutes daily for 28 consecutive days. The device is a handheld electrical pulse generator and a pair of electrodes will be placed on the skin for stimulation. The electrical pulse generator is turned on and the amplitude of stimulation increased to the greatest amount tolerated, however, the vagus nerve will not be stimulated. Patients will be followed through day 57 to assess durability of the intervention.
Treatment groups
Trial outcomes
Primary outcomes
Change in Musculoskeletal Pain From Baseline.
Patients rate their musculoskeletal pain by making a mark on a 10cm anchored Visual Analog Scale where 0=no musculoskeletal pain and 10 =worst possible musculoskeletal pain.
Secondary outcomes
Percentage of Subjects With Treatment Emergent Adverse Events.
The percentage of participants with grade 2 or higher or specific grade 1 treatment emergent adverse events will be assessed using the NCI-CTAEversion4. The percentage of participants with grade 2 or higher treatment emergent adverse events will be assessed using the NCI-CTAEversion4. The percentage of participants with grade 2 or higher treatment emergent adverse events will be assessed using the NCI-CTAEversion4.
Change in Musculoskeletal Pain From Baseline
Patients rate their musculoskeletal pain by making a mark on a 10cm anchored Visual Analog Scale where 0=no musculoskeletal pain and 10 =worst possible musculoskeletal pain.
Change in Musculoskeletal Pain From Baseline
Patients rate their musculoskeletal pain by making a mark on a 10cm anchored Visual Analog Scale where 0=no musculoskeletal pain and 10 =worst possible musculoskeletal pain.
Change in Musculoskeletal Pain From Day 29 to Day 57
Patients rate their musculoskeletal pain by making a mark on a 10cm anchored Visual Analog Scale where 0=no musculoskeletal pain and 10 =worst possible musculoskeletal pain.
Other outcomes
Functional gut permeability
Functional gut permeability is assessed by measuring urinary C mannitol/lactulose ratios after oral injestion of the sugars, in timed urine collections over 24 hours.
Stool microbiome
Stool microbiome is assessed by determining changes in metagenomics, metatranscriptomics and metabolomics from baseline in stool specimens.
Gut permeability: serum I-FABP, zonulin and LPS, and by stool calprotectin and zonulin
Gut permeability: serum I-FABP is determined by measuring levels of serum I-FABP
Gut permeability: zonulin
Gut permeability: zonulin is determined by measuring levels of serum zonulin
Sponsors and contacts
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Northwell Health
Lead sponsor
Lupus Research Alliance
Collaborator