STEM-Parkinson's Disease

Sponsor

Scion NeuroStim (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04797611

Collaborator

(none)

218

Enrollment

Locations

Arms

11.4

Anticipated Duration (Months)

12.8

Patients Per Site

1.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a double-blinded, controlled, randomized clinical trial (RCT) to establish the safety and efficacy of a non-invasive neuromodulation device for treating symptoms associated with Parkinson's disease.

Condition or Disease	Intervention/Treatment	Phase
Parkinson Disease Parkinson's Disease and Parkinsonism	Device: Non-invasive brainstem stimulation	N/A

Detailed Description

Up to 218 participants will enter the double-blinded, controlled, randomized clinical trial (RCT) and will self-administer treatments twice daily in the home setting over a period of 12 weeks following a 4 week baseline period. The RCT will have 6 study visits: 3 visits at the study center and 3 visits completed in the participant's home by video call.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

218 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Triple (Participant, Care Provider, Investigator)

Primary Purpose:

Treatment

Official Title:

Non-Invasive Brainstem Modulation for the Treatment of Non-Motor Symptoms in Parkinson's Disease: A Randomized Controlled Trial (RCT)

Actual Study Start Date :

May 19, 2022

Anticipated Primary Completion Date :

May 1, 2023

Anticipated Study Completion Date :

May 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Other: Investigational Treatment 1 Investigational treatment mode (stimulation pattern) 1	Device: Non-invasive brainstem stimulation Study participants will self-administer ~19-minute treatments twice daily in the home setting over 12 weeks using a non-invasive brainstem modulation device. The device has been deemed to be a nonsignificant risk for studies in Parkinson's disease by the United States Food and Drug Administration.
Other: Investigational Treatment 2 Investigational treatment mode (stimulation pattern) 2	Device: Non-invasive brainstem stimulation Study participants will self-administer ~19-minute treatments twice daily in the home setting over 12 weeks using a non-invasive brainstem modulation device. The device has been deemed to be a nonsignificant risk for studies in Parkinson's disease by the United States Food and Drug Administration.

Arm

Intervention/Treatment

Other: Investigational Treatment 1

Investigational treatment mode (stimulation pattern) 1

Device: Non-invasive brainstem stimulation

Study participants will self-administer ~19-minute treatments twice daily in the home setting over 12 weeks using a non-invasive brainstem modulation device. The device has been deemed to be a nonsignificant risk for studies in Parkinson's disease by the United States Food and Drug Administration.

Other: Investigational Treatment 2

Investigational treatment mode (stimulation pattern) 2

Device: Non-invasive brainstem stimulation

Outcome Measures

Primary Outcome Measures

Change from baseline in The International Parkinson and Movement Disorder Society - Non-Motor Rating Scale (MDS-NMS) Total Score [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. Scores range between 0-832, with the higher score indicating greater non-motor symptom burden. (day 113 score - average of baseline scores from days 1 and 29)

Secondary Outcome Measures

Change from baseline in The International Parkinson and Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Total score (Sum of Parts I, II and III) [3 months]
The MDS- UPDRS is a multimodal scale consisting of four parts. Part I assesses non-motor aspects of experiences of daily living and consists of two components (range 0-52). Part II is a 13- item patient-reported assessment of motor aspects of experiences of daily living. Scores range between 0-52. Part III assesses motor signs of Parkinson's disease and is completed by the rater (range of 0-132). Part III consists of 33 scores based on 18 items. In all parts, higher scores indicate more symptoms. The range for the MDS-UPDRS total (sum of parts I, II and III) is 0-236. (baseline - week 12)
Change from baseline in The International Parkinson and Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II [3 months]
The MDS-UPDRS Part II is a 13-item patient-reported assessment of motor aspects of experiences of daily living. Scores range between 0-52, with the higher score indicating greater impairment to activities of daily living. (day 113 score - average of baseline scores from days 1 and 29)
Change from baseline in the Clinical Global Impression-Improvement (CGI-I) [3 months]
The CGI-I is a clinician assessment the extent of clinically meaningful change that has occurred in the patient's illness at day 113 relative to a baseline state (assessed at day 29). Changes in all aspects of Parkinson's disease (e.g., motor symptoms, non-motor symptoms and complications of anti-Parkinsonian medications) are considered.
Change from baseline in the MDS-UPDRS Part III [3 months]
The MDS-UPDRS Part III is a 33-item assessment of motor function evaluated by a trained blinded rater. Scores range between 0-132 with higher scores indicating more sever motor symptoms. (day 113 score - average of baseline scores from days 1 and 29)
Change from baseline in the Parkinson's Disease Quality of Life Questionnaire Summary Index (PDQ-39 SI) [3 months]
The PDQ-39 SI is a 39-item patient-reported quality of life measure that assesses how often people living with PD are affected across 8 dimensions of daily living. Scores range from 0-100, with the higher scores indicating worse perception of quality of life. (day 113 score - average of baseline scores from days 1 and 29)

Other Outcome Measures

Change from baseline in the Mini-Balance Evaluation Systems Test [3 months]
a measure of dynamic balance, functional mobility, and gait. This measure will be evaluated as a safety outcome to assess whether device therapy negatively impacts balance or gait for participants with Parkinson's disease (day 113 score - baseline day 29 score).
Change from baseline in the Montreal Cognitive Assessment [3 months]
a screening instrument used to facilitate the assessment of cognitive impairment.
Change from baseline in the Oral Symbol Digit Modality Test [3 months]
a brief and commonly used test to evaluate processing speed.
Change from baseline in the Parkinson's Disease Sleep Scale 2 [3 months]
an assessment to quantify nocturnal sleep issues in Parkinson's disease.
Change from baseline in Epworth Sleepiness Scale [3 months]
a brief measure that is commonly used to assess daytime sleepiness.
Change from baseline in the Parkinson Anxiety Scale [3 months]
a brief questionnaire to detect anxiety severity in Parkinson's disease.
Change from baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue Scale [3 months]
a measure of an individual's level of fatigue during their usual daily activities over the past week.
Change from baseline in the Geriatric Depression Scale-15 [3 months]
a short questionnaire for assessing depression in older adults.
Change from baseline in the MDS-NMS Non-Motor Fluctuations [3 months]
a rater completed assessment evaluating fluctuations of non-motor symptoms in Parkinson's disease.
Change from baseline in the Unified Parkinson's Disease Rating Scale Part IV [3 months]
an assessment of complications of anti-Parkinsonian therapies.
Change from baseline in a Non-Motor Symptom focused Clinical Global Impression-Improvement [3 months]
clinician assessment the extent of clinically meaningful change that has occurred as it relates to the patient's non-motor symptoms.
Change from baseline in the Zarit Burden Interview [3 months]
measure of caregiving burden completed by caregivers.
Change from baseline in the Patient Reported Outcome - Parkinson's Disease [3 months]
a self-rating tool to assess symptom severity in Parkinson's disease.
Change from baseline in The Modified Schwab and England Activities of Daily Living Scale [3 months]
a clinical outcome assessment of an individual's ability to function independently in activities of daily living.
Change from baseline in EncephaLogTM 3 meter Timed Up and Go Test [3 months]
smart phone application assessment that provides indication of risk of falls in adults.
Change from baseline in EncephaLogTM finger tapping test [3 months]
smart phone application providing a quantitative measure of bradykinesia.
Change from baseline in EncephaLogTM 10 meter Timed Up and Go Test [3 months]
smart phone application that measures gait and that provides indication of risk of falls in adults.
Change from baseline in Unified Parkinson's disease Rating Scale Part I (UPDRS I) [3 months]
a measure of mentation, behavior, and mood in Parkinson's disease.
Change from baseline in Hoehn & Yahr (H&Y) [3 months]
staging tool describing the level of disability in Parkinson's disease.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 85 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Adult participants (aged 18 - 85 years inclusive)
Diagnosed with Parkinson's disease according to the UK Brain Bank Criteria
Demonstrates a positive response to oral anti-Parkinsonian medications (i.e. dopamine replacement therapies) and treated with these medications for minimum of three years prior to the screening visit
Report limitation to 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.
Anticipate being able to remain on a stable regimen of medications used for the management of PD motor and non-motor symptoms and not to introduce new medications used to treat symptoms associated with PD during the trial.
Fully vaccinated from COVID-19 (at least 2 weeks from their final dose) with one of the current World Health Organization evaluated vaccines, prior to Screen.
Have at minimum a moderate burden of non-motor symptoms associated with Parkinson's disease
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.
Have capabilities to use and access smartphones and or tablets for the collection of some study data.
Must be willing to answer questions related to sexual interest, arousal, and performance in an interview with study staff.

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 (i.e., are not yet 3 years removed from their first menopausal symptom), 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 evidence of dementia at study screen.
Have experienced a myocardial infarction, angina, or stroke within the past 12 months.
Are receiving deep brain stimulation therapy.
Are treated with a pump for continuous delivery of dopamine replacement medication.
Use apomorphine rescue.
Have received MRI guided high intensity focused ultrasound within the past 12 months.
Experience frequent falls.
Work night shifts
Use a hearing aid that is implanted or that cannot be easily removed and replaced.
Have a cochlear implant.
Have chronic (>3 months) tinnitus.
Have previously been diagnosed with traumatic brain injury with ongoing sequalae.
Have been diagnosed with a co-morbid neurological disorder that may present with symptoms overlapping with PD (e.g., stroke, brain tumor, epilepsy, Alzheimer's disease, multiple sclerosis, amyotrophic lateral sclerosis, atypical Parkinsonism or aneurysm)
Have unresolved complications from a previous surgical procedure at the baseline visits, such as swelling or persistent pain, that requires medical intervention.
Have active ear infections, or other significant ear problems.
Have a recent history of frequent ear infections (≥ 1 per year over the past two years)
Are currently enrolled or have participated in another interventional clinical trial within the last 30 days.
Have had eye surgery within the previous three months or ear surgery within the previous six months.
Have planned surgery scheduled to occur during the study that requires sedation and/or would typically be followed with a prescription for pain management.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Movement Disorder Center of Arizona	Scottsdale	Arizona	United States	85258
2	Cedars-Sinai Medical Center	Los Angeles	California	United States	90048
3	CenExel Rocky Mountain Clinical Research	Englewood	Colorado	United States	80113
4	Parkinson's Disease and Movement Disorders Center of Boca Raton	Boca Raton	Florida	United States	33486
5	Headlands Research Orlando	Orlando	Florida	United States	32819
6	USF Parkinson's Disease and Movement Disorders Center	Tampa	Florida	United States	33613
7	University of Kansas Medical Center - Parkinson's Disease Center	Kansas City	Kansas	United States	66160
8	Quest Research	Farmington Hills	Michigan	United States	48334
9	Mercy Health Saint Mary's	Grand Rapids	Michigan	United States	49503
10	University of New Mexico	Albuquerque	New Mexico	United States	87131
11	Meridian Clinical Research	Raleigh	North Carolina	United States	27607
12	Cleveland Clinic	Cleveland	Ohio	United States	44195
13	Thomas Jefferson University	Philadelphia	Pennsylvania	United States	19107
14	Medical University of South Carolina	Charleston	South Carolina	United States	29425
15	Veracity Neuroscience	Memphis	Tennessee	United States	38157
16	Houston Methodist Neurological Institute	Houston	Texas	United States	77030
17	Riverside Neurology Specialists	Newport News	Virginia	United States	23601