Pre-Gene Therapy Study in Parkinson's Disease and Multiple System Atrophy

Study Overview
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Brief Summary
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The objective of this study is to describe disease progression in study participants diagnosed with early Parkinson's Disease or Multiple System Atrophy - Parkinsonian Type up to 18 months as delineated by clinical and biochemical parameters.

Official Title
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A Pre-Gene Therapy Study of Early Parkinson's or Multiple System Atrophy Progression by Longitudinal Clinical and Biomarker Assessments

Conditions
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Parkinson's Disease, Multiple System Atrophy, Parkinson Variant, Multiple System Atrophy

Intervention / Treatment
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* Other: This is an observational study.


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of Idiopathic PD Diagnosis of MSA-P One to six years from clinical diagnosis of PD Less than 4 years from clinical diagnosis of MSA with expected survival >3 years Stable medication regimen Ability to walk with or without an assistive device Exclusion Criteria: Movement disorder due to known cerebrovascular disease, brain tumor, trauma, exposure to parkinsonian-linked toxicants or other neurological diseases Presence of dementia, psychosis, substance abuse or poorly controlled depression Prior brain surgery (i.e. deep brain stimulation) or other brain imaging abnormalities History of cancer or poorly controlled medical conditions Receiving an investigational drug NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Ages Eligible for Study
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Minimum Age: 35 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Early Parkinson's disease<br> | Other: This is an observational study.<br>* This is an observational study.<br>|
| Possible or Probable MSA-P<br> | Other: This is an observational study.<br>* This is an observational study.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Change from baseline in the MDS-UPDRS. The MDS-UPDRS contains 4 subscales: Part I, non-motor aspects of experiences of daily living (13 items); Part II, motor aspects of experiences of daily living (13 items); Part III, motor examination (33 scores based on 18 items); Part IV, motor complications (6 items). The rating for each item, or sub-item, is from 0 (normal) to 4 (severe). The total score for each Part is obtained from the sum of the corresponding item scores. | 18 months | 
| MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS) | Change from baseline in the Unified Multiple System Atrophy Rating Scale (UMSARS) and compared to placebo. UMSARS total scores range from 0-104 points with higher scores indicating greater severity of impairment. | 18 Months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in the quality of life as measured by Parkinson's Disease Questionnaire (PDQ-39) | Change from baseline in the Parkinson's Disease Questionnaire (PDQ-39). PDQ-39 is a self-reported 39-item questionnaire designed to measure health related quality of life in Parkinson's Disease patients. It provides scores in 8 domains: mobility, activities of daily living, emotions, stigma, social support, cognitive function, bodily discomfort and communication, as well as a summary index score for quality of life. | 18 months | 
| Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL) | Change from baseline in the Multiple System Atrophy Quality of Life (MSA-QoL) scale. MSA-QoL is a self-reported questionnaire that measures MSA impact in day to day activities. Scale consists of 40 items with a five response option format (0 - no problem to 4 extreme problem) and a not applicable response option. | 18 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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MSA, MSA-P, PD, Observational, Natural History