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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Parkinson's Disease; Multiple System Atrophy; Multiple System Atrophy, Parkinson Variant
• Intervention / Treatment: Other: This is an observational study.

• Study Type: Observational
• Enrollment (Estimated): 150

Contacts and Locations

Study Locations

• Poland
  • Katowice, Poland
    • Specjalistyczna Praktyka Lekarska, Prof. Grzegorz Opala
  • Warsaw, Poland
    • Mazowiecki Szpital Bródnowski w Warszawie, Oddział Neurologii
  • Warsaw, Poland
    • Samodzielny Publiczny Szpital Kliniczny im. prof. Witolda Orłowskiego, Oddział Neurologii i Epileptologii
  • Lesser Poland Voivodeship
    • Krakow, Lesser Poland Voivodeship, Poland
      • Zespol Poradni Specjalistycznych - Botaniczna 3 Poradnia Neurologiczna Szpital Uniwersytecki w Krakowie
• United States
  • California
    • Irvine, California, United States, 92697
      • University of California Irvine
  • Florida
    • Boca Raton, Florida, United States, 33486
      • Parkinson's Disease and Movement Disorders Center of Boca Raton
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
  • Michigan
    • Farmington Hills, Michigan, United States, 48334
      • QUEST Research Institute
  • Ohio
    • Columbus, Ohio, United States, 43214
      • Ohio Health Research Institute
  • Oregon
    • Portland, Oregon, United States, 97239
      • OHSU Parkinson Center & Movement Disorders Program
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt University Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 35 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants will have a diagnosis of early Parkinson's disease OR possible or probably MSA-P

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Early Parkinson's disease	Other: This is an observational study.; This is an observational study.
Possible or Probable MSA-P	Other: This is an observational study.; This is an observational study.

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: AskBio Inc

Study record dates

Study Major Dates

• Study Start (Actual): September 29, 2022
• Primary Completion (Estimated): November 1, 2025
• Study Completion (Estimated): November 1, 2025

Study Registration Dates

• First Submitted: October 13, 2022
• First Submitted That Met QC Criteria: January 17, 2023
• First Posted (Actual): January 26, 2023

Study Record Updates

• Last Update Posted (Estimated): October 2, 2025
• Last Update Submitted That Met QC Criteria: September 29, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: MSA; Natural History; Observational; PD; MSA-P
• Additional Relevant MeSH Terms: Synucleinopathies; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; Movement Disorders; Parkinsonian Disorders; Basal Ganglia Diseases; Primary Dysautonomias; Autonomic Nervous System Diseases; Parkinson Disease; Multiple System Atrophy
• Other Study ID Numbers: ASK-PD-0-CS-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No