PARKINSON DISEASE (Disorder)
Conditions
Keywords
Parkinsonian Disorders, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Parkinson Disease, Tremor, Movement Disorder
Brief summary
Vista PD is a randomized, placebo-controlled, multicenter study to evaluate the efficacy, PK, safety, and tolerability of VIM0423 in adults with Parkinson's disease tremor insufficiently responsive to dopaminergic therapy. The main objectives of this clinical trial are to determine the following: * Does VIM0423 therapy improve tremor symptoms in adults with Parkinson's disease? * Is VIM0423 well tolerated in individuals with Parkinsons's disease? and * Do the therapeutic effects of VIM0423 confer improvements on daily function and quality of life?
Detailed description
Parkinson's disease (PD) is the second most common neurodegenerative condition, with studies estimating US prevalence among individuals aged 45 years and older rising to approximately 1,238,000 by 2030. More than 75% of individuals with PD will experience resting tremor at some point during their disease course, and approximately 60% additionally suffer from a symptomatic tremor during action or movement, complicating functional tasks. The distress caused by PD tremor is profound, with individuals consistently identifying it as their "most bothersome" symptom in early disease, and more impactful than other motor and nonmotor symptoms. Despite the clear importance of this symptom to patient experience, significant unmet need in tremor management persists. Tremor-specific efficacy of dopaminergic therapy, the mainstay of early PD pharmacology, is often inconsistent or incomplete, giving rise to the clinical concept of "dopamine-responsive" versus "dopamine-refractory" tremor. Anti-tremor efficacy of anticholinergic agents has been recognized for decades; however, their clinical utility is limited by both central and peripheral adverse effects VIM0423 is being developed as an oral medication designed to improve the central and peripheral tolerability seen with other anticholinergic medications. Vista PD (Study VIM0423-251) is a Phase 2, multicenter study designed to assess the efficacy, PK, safety, and tolerability of VIM0423 in adults with Parkinson's disease tremor insufficiently responsive to dopaminergic therapy. Up to 80 individuals will be enrolled in the trial. Cohort 1 will be comprised of adults diagnosed with PD who are on stable and adequate dopaminergic therapy (oral levodopa or oral dopamine agonist only), with residual tremor. Participants in Cohort 1 will be randomized (1:1) to receive either VIM0423 or matching placebo (up to 5 pills a day\[MO1.1\]\[TG1.2\]\[MO1.3\], taken once daily) for 7 weeks. The Investigator and Participant will not know whether \[MO2.1\]patients are receiving VIM0423 or placebo, but that information will be available if needed. Cohort 2 will be comprised of adults diagnosed with PD who are dopaminergic naïve, as defined by: no prior or ongoing usage of levodopa (in any form) or dopamine agonist (in any form), and no anticipated plans to initiate said therapies for the duration of the study. Participants in Cohort 2 will be randomized (1:1) to receive either VIM0423 or matching placebo (up to 5 pills a day\[MO3.1\], taken once daily) for 7 weeks. The Investigator and Participant will not know whether patients are receiving VIM0423 or placebo, but that information will be available if needed. The total time of participation in the trial is up to 12 weeks and requires 6 in person visits. The study schedule includes Screening and Baseline (up to 4 weeks, 2 visits), Treatment period (including taper, 7 weeks, 3 Visits), and Safety follow-up (1 week, 1 Visit). Assessments of changes in Parkinson's disease will be made by study personnel. Clinical labs, EKGs, and Adverse events will be monitored throughout the study. Participants will be asked to use a wearable device during the study and perform self-assessments of their Parkinson's tremor and its impact on their activities of daily living.
Interventions
DRUGVIM0423
VIM0423 is a combination drug product containing two active ingredients (VMA-1001 and VMA-1002)
DRUGVIM0423 Placebo
Matching VIM0423 placebo product containing no active ingredient
Sponsors
Vima Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Masking description
The study sponsor, participants, the CRO, Investigator, and all site personnel (except pharmacists and pharmacy staff) will be blinded to treatment assignments until the database has been locked and restricted from further edits.
Intervention model description
Parallel cohorts of participants receiving active or placebo drug products
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Participant must be male or nonpregnant female between 18 and 65 years of age (inclusive) at Visit 1 (Screening). * Diagnosis of clinically probable or clinically established idiopathic Parkinson's disease (PD) at Visit 1 (Screening) established by the MDS 2015 criteria which must include tremor as one of the cardinal characteristics. * Cohort 1: Currently using levodopa (any oral form only) or oral dopamine agonist at a stable dose and regimen of at least 10 weeks prior to Visit 1 (Screening); with no anticipated changes to said therapies for the duration of the study; and on a minimum of 300 mg LEDD. Cohort 2: Individuals who are dopaminergic naïve, as defined by: no prior or ongoing usage of levodopa (in any form) or dopamine agonist (in any form), and no anticipated plans to initiate said therapies for the duration of the study. * The participants must meet protocol-specified requirements for baseline tremor scores.
Exclusion criteria
* Currently (within 7 days of Screening) taking any anticholinergic medication. * Prior deep brain stimulation (DBS) or any other form of invasive neurosurgical intervention (including, but not limited to, magnetic resonance-guided focused ultrasound thalamotomy, ablative thalamotomy, gamma knife thalamotomy) Other protocol-specified inclusion and
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | From baseline to Week 6 | The MDS-UPDRS is a multi-part assessment designed to evaluate various aspects of PD and its impact on the individual, including motor and non-motor symptoms. There are four parts, all of which will be completed in this study: Part I (non-motor experiences of daily living), Part II (motor experiences of daily living), Part III (motor examination), and Part IV (motor complications). Each part has varying maximum scores. In all cases, a higher score represents a more severe status. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in Clinical Global Impression of Severity (CGI-S) as assessed by the clinician | From baseline to Week 6 | The CGI-S assesses the clinician's impression of the participant's current illness state. The clinician should use his/her total clinical experience with this population and rate the current severity of the participant's PD on a scale from 1 (normal, not at all ill) to 7 (among the most extremely ill). |
| Change from baseline in Tremor Research Group Essential Tremor Rating Scale - Activities of Daily Living (TETRAS-ADL) | From baseline to Week 6 | The TETRAS-ADL is a standardized assessment of the impact of tremor on activities of daily living in individuals with essential tremor. There are 12 items: speaking; feeding with a spoon; drinking from a glass; hygiene; dressing; pouring; carrying food on trays, plates or similar items; using keys; writing; working; overall disability; social impact. Each item is scored 0 (normal) to 4 (severe/severely abnormal), for a total possible score of 48, with a higher score indicated a worse impact of tremor. |
| Clinical Global Impression of Change (CGI-C) | From baseline to Week 6 | The CGI-C assesses the clinician's impression of how the participant's current illness has changed relative to baseline/pre-study. The clinician should use his/her total clinical experience with the individual participant and rate the participant's change in signs and reported symptoms using the scale: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), or 7 (very much worse). |
| Patient Global Impression of Change (PGI-C) | From baseline to Week 6 | The PGI-C is a written scale which will be used to solicit the participant's self-report of overall difference in PD related symptoms compared to his/her pre-study baseline. The score for each timepoint will be: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), or 7 (very much worse). |
| Change from baseline in Scales for Outcomes in Parkinson's Disease (SCOPA) - Sleep | From baseline to Week 6 | The SCOPA-Sleep is a validated patient-completed scale for assessing various components of sleep in individuals with PD: use of medication for sleep (2 items), nighttime sleep (5 items), global evaluation of sleep (1 item), and daytime sleepiness (6 items). A higher score represents a worse outcome. |
Countries
Canada, United States
Contacts
CONTACTStudy Director
Outcome results
None listed