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

NCT05699460 | Active Not Recruiting DMC

• 1 other identifier: ASK-PD-0-CS-001
• Study Type: observational
• Target: 150
• Locations: 2 countries
• Sites: 11

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.

Conditions

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

Keywords

MSA; MSA-P; PD; Observational; Natural History

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (2)

• Change in the quality of life as measured by Parkinson's Disease Questionnaire (PDQ-39)

  18 months

• Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)

  18 months

Study Arms (2)

Early Parkinson's disease

Other: This is an observational study.

Possible or Probable MSA-P

Other: This is an observational study.

Interventions

This is an observational study. OTHER

This is an observational study.

Early Parkinson's disease; Possible or Probable MSA-P

Eligibility Criteria

• Age: 35 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• AskBio Inc: lead

Study Sites (11)

University of California Irvine

Irvine, California, 92697, United States

Location

Parkinson's Disease and Movement Disorders Center of Boca Raton

Boca Raton, Florida, 33486, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Quest Research Institute

Farmington Hills, Michigan, 48334, United States

Location

Ohio Health Research Institute

Columbus, Ohio, 43214, United States

Location

OHSU Parkinson Center & Movement Disorders Program

Portland, Oregon, 97239, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Zespol Poradni Specjalistycznych - Botaniczna 3 Poradnia Neurologiczna Szpital Uniwersytecki w Krakowie

Krakow, Lesser Poland Voivodeship, Poland

Location

Specjalistyczna Praktyka Lekarska, Prof. Grzegorz Opala

Katowice, Poland

Location

Mazowiecki Szpital Bródnowski w Warszawie, Oddział Neurologii

Warsaw, Poland

Location

Samodzielny Publiczny Szpital Kliniczny im. prof. Witolda Orłowskiego, Oddział Neurologii i Epileptologii

Warsaw, Poland

Location

MeSH Terms

Conditions

Parkinson Disease; Multiple System Atrophy

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Primary Dysautonomias; Autonomic Nervous System Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 13, 2022
• First Posted: January 26, 2023
• Study Start: September 29, 2022
• Primary Completion: November 1, 2025
• Study Completion: November 1, 2025
• Last Updated: October 2, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (7); PL (4)