Non-Invasive Brainstem Modulation for the Treatment of Non-Motor Symptoms in Parkinson's Disease: An Open Label Extension (OLE) Study
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Parkinson Disease
- Sponsor
- Scion NeuroStim
- Enrollment
- 148
- Locations
- 15
- Primary Endpoint
- Change From Baseline in The International Parkinson and Movement Disorder Society - Non-Motor Rating Scale (MDS-NMS) Total Score
- Status
- Terminated
- Last Updated
- 10 months ago
Overview
Brief Summary
This study seeks to establish the safety and efficacy of extended twice daily time-varying caloric vestibular stimulation treatments for treating symptoms associated with Parkinson's disease. Only participants who completed the STEM-PD randomized controlled trial portion (NCT04797611) are eligible to participate in the open label extension portion.
Detailed Description
Up to 220 participants will enter an open label extension study during which all study participants will receive active (i.e., time-varying caloric vestibular stimulation) treatment for 12 weeks (84 days). Study participants will be followed for 16 weeks (112 days) post treatment-cessation and then the twice daily active treatments will be re-introduced for the final 8 weeks (56 days).
Investigators
Eligibility Criteria
Inclusion Criteria
- •Completion of the study activities in the STEM-PD RCT trial (see STEM-PD RCT eligibility criteria noted below).
- •Participants must be willing and able to give consent to participate in the study trial.
- •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 first three months of the OLE (Day197).
Exclusion Criteria
- •Participant anticipates being unable to attend all visits and complete all study activities.
- •Has a planned surgery scheduled to occur during the first 90 days of the OLE that would typically be followed with a prescription for pain management
- •STEM-PD RCT Eligibility criteria:
- •Adult participants (aged 18 - 85 years inclusive).
- •Have been diagnosed with PD according to the UK Brain Bank Criteria (allowing for an exclusion in Step 2 for "more than one affected relative").
- •Participants must have demonstrated a sustained positive response to 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 reports limitation 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.).
- •Participants must be able and willing to consent to participate in the study for the RCT and OLE.
- •Participants must be willing and able to comply with study requirements.
- •Participants must have at minimum a moderate burden of NMS (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).
Outcomes
Primary Outcomes
Change From Baseline in The International Parkinson and Movement Disorder Society - Non-Motor Rating Scale (MDS-NMS) Total Score
Time Frame: 3 months (NCT04797611 RCT: Day 29 - Day 113; This OLE: Day 113 to Day 197)
The primary endpoint is the change in MDS-NMS total score during the first treatment period during the open label extension (OLE) relative to the score at the end of the NCT04797611 randomized controlled trial (RCT) treatment period (day 113) for the passive-active treatment group compared to the change in MDS-NMS total score during the RCT treatment period relative to the pretreatment baseline. The MDS-NMS is 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. 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.
Secondary Outcomes
- Change From Baseline in The International Parkinson and Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II(3 months (NCT04797611 RCT: Day 29 - Day 113; this OLE: Day 113 to Day 197))
- Change From Baseline in the Parkinson's Disease Quality of Life Questionnaire Summary Index (PDQ-39 SI)(3 months (NCT04797611 RCT: Day 29 - Day 113; this OLE: Day 113 to Day 197)
- Change From Baseline in the Combined Measure of The International Parkinson and Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Parts I, II and III(3 months (NCT04797611 RCT: Day 29 - Day 113; this OLE: Day 113 to Day 197)
- Change From Baseline in the Clinical Global Impression - Improvement (CGI-I)(RCT: from Baseline (Day 29) up to 3 months (Day 113); OLE: from Baseline (Day 29) up to 11 months (Day 365))
- Change From Baseline in the International Parkinson and Movement Disorder Society Unified Parkinson's Disease Rating Scale Part III: Motor Examination (MDS-UPDRS Part III)(3 months (NCT04797611 RCT: Day 29 - Day 113; this OLE: Day 113 to Day 197))