Clinical trial NCT05699460: Pre-Gene Therapy Study in Parkinson's Disease and Multiple System Atrophy
Official title: A Pre-Gene Therapy Study of Early Parkinson's or Multiple System Atrophy Progression by Longitudinal Clinical and Biomarker Assessments
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Parkinson's Disease
Multiple System Atrophy, Parkinson Variant
Multiple System Atrophy
Keywords: MSA
MSA-P
PD
Observational
Natural History
Lead sponsor: AskBio Inc
Sponsor class: INDUSTRY
First posted: 2023-01-26
Enrollment: 150
Sex: ALL
Minimum age (years): 35
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants will have a diagnosis of early Parkinson's disease OR possible or probably MSA-P

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
timeFrame: 18 months
measure: MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)
timeFrame: 18 Months
Secondary outcomes:
measure: Change in the quality of life as measured by Parkinson's Disease Questionnaire (PDQ-39)
timeFrame: 18 months
measure: Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)
timeFrame: 18 months
Other outcomes:
Locations:
city: Irvine
country: United States
facility: University of California Irvine
state: California
city: Boca Raton
country: United States
facility: Parkinson's Disease and Movement Disorders Center of Boca Raton
state: Florida
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Farmington Hills
country: United States
facility: Quest Research Institute
state: Michigan
city: Columbus
country: United States
facility: Ohio Health Research Institute
state: Ohio
city: Portland
country: United States
facility: OHSU Parkinson Center & Movement Disorders Program
state: Oregon
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Kraków
country: Poland
facility: Zespol Poradni Specjalistycznych - Botaniczna 3 Poradnia Neurologiczna Szpital Uniwersytecki w Krakowie
state: Lesser Poland Voivodeship
city: Katowice
country: Poland
facility: Specjalistyczna Praktyka Lekarska, Prof. Grzegorz Opala
city: Warszawa
country: Poland
facility: Mazowiecki Szpital Bródnowski w Warszawie, Oddział Neurologii
city: Warszawa
country: Poland
facility: Samodzielny Publiczny Szpital Kliniczny im. prof. Witolda Orłowskiego, Oddział Neurologii i Epileptologii
Study officials:
References: