Non-Invasive Brainstem Modulation for the Treatment of Non-Motor Symptoms in Parkinson's Disease: A Randomized Controlled Trial (RCT)
Overview
- Phase
- N/A
- Intervention
- Not specified
- Conditions
- Parkinson Disease
- Sponsor
- Scion NeuroStim
- Enrollment
- 218
- Locations
- 23
- Primary Endpoint
- Change in The International Parkinson and Movement Disorder Society Non-Motor Rating Scale (MDS-NMS) Total Score
- Status
- Completed
- Last Updated
- 11 months ago
Overview
Brief Summary
This is a double-blinded, controlled, and randomized clinical trial (RCT) to establish the safety and efficacy of a non-invasive neuromodulation device for treating symptoms associated with Parkinson's disease.
Detailed Description
The double-blinded, controlled, randomized clinical trial (RCT) will be conducted at 15 centers, at minimum, in the United States and the United Kingdom with the majority of centers in the United States. Up to 184 participants will enter and will self-administer treatments twice daily in their home setting over a period of 12 weeks following a 4 week baseline period. Each participant will complete 6 study visits: 3 visits at the study center and 3 visits completed in the participant's home by video call.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Adult participants (aged 18 - 85 years inclusive)
- •Diagnosed with Parkinson's disease according to the UK Brain Bank Criteria (allowing for an exclusion in Step 2 for "more than one affected relative").
- •Demonstrates a sustained, positive response to dopamine replacement therapies (DRTs) (e.g., levodopa, dopamine agonists or monoamine oxidase inhibitors) defined as either good or excellent responses (50-100%) for at least one year or moderate responses (30-49)% for at least three years prior to screen.
- •Participant-reported or clinician-investigator-determined limitation, based on knowledge of medical history, to one or more activities of daily living (e.g., writing, walking, bathing, dressing, eating, toileting, etc.)
- •Able and willing to consent to participate in the study.
- •Willing and able to comply with study requirements.
- •Participants and investigators must expect that the participant will be able to remain on a stable regimen of concomitant therapies used for the management of PD motor and non-motor symptoms and not to introduce new medications used to treat motor or non-motor symptoms associated with PD during the study.
- •Have at minimum a moderate burden of non-motor symptoms associated with Parkinson's disease (i.e., MDS-UPDRS part Ia and part Ib summed total score ≥ 9) at study screen to avoid floor effects for the primary endpoint (MDS-NMS).
- •The principal investigator or designee must have confidence in the participant's ability to reliably use the TNM™ device, understand the assessments (provided in English only) and to complete the assessment battery within a given on-state period.
- •Have a study partner (defined as someone who sees the participant for more than one hour a day, 3 times per week) that is willing to consent and participate in the trial.
Exclusion Criteria
- •Participant anticipates being unable to attend all visits and complete all study activities.
- •Women of child-bearing potential who are pregnant or plan to become pregnant during the course of the study trial. Women of child-bearing potential , who are not abstinent or exclusively in same sex relationships must:
- •Test negative for pregnancy as indicated by a negative urine pregnancy test
- •Agree to use an approved contraception method for the entirety of the trial
- •Have a history or prior diagnosis of dementia or or adjusted score ≤ 20 on the Montreal Cognitive Assessment (MoCA) at the screening visit
- •Have experienced a myocardial infarction, angina, or stroke within the past 12 months, or a transient ischemic attack within the past 6 months.
- •Are receiving deep brain stimulation therapy.
- •Are treated with a pump for continuous delivery of dopamine replacement medication.
- •Have received MRI guided high intensity focused ultrasound within the past 12 months.
- •Experience frequent falls. (defined as 2 or more falls in the past month related to Parkinson's disease). Parkinson's falls are defined as falls associated with bradykinesia, freezing, turning, change in posture and postural dizziness and do not include accidental falls.
Outcomes
Primary Outcomes
Change in The International Parkinson and Movement Disorder Society Non-Motor Rating Scale (MDS-NMS) Total Score
Time Frame: 3 months
The MDS-NMS is a 52-item rater-administered scale used to assess a wide range of non-motor symptoms in Parkinson's disease. It measures both frequency (0/never to 4/ \>51% of the time) and severity (0/not present to 4/major distress or disturbance) of 13 domains (depression, anxiety, apathy, psychosis, impulse control/related disorders, cognition, orthostatic hypotension, urinary, sexual, gastrointestinal, sleep/wakefulness, pain, and other). Each question is scored by multiplying frequency x severity. All question scores for each domain are summed, and the scores for each domain are summed to provide the Total Score (range = 0-832) with the higher score indicating greater non-motor symptom burden. The higher the total score, the more progressed (i.e., worse) is the disease state. The change/difference in scores between the baseline (average of Days 1 and 29) and end of study treatment (Day 113) are reported. A positive change in scores indicates worsening.
Secondary Outcomes
- Change in The International Parkinson and Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Parts I, II, and III Combined Score(3 months)
- Change in The International Parkinson and Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II(3 months)
- Change From Baseline in the Clinical Global Impression-Improvement (CGI-I)(3 months)
- Change in the International Parkinson and Movement Disorder Society Unified Parkinson's Disease Rating Scale MDS-UPDRS Part III(3 months)
- Change From in the Parkinson's Disease Quality of Life Questionnaire Summary Index (PDQ-39 SI)(3 months)