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Completed

NCT Number: NCT05699460

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

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.

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Key information

Conditions

Parkinson's Disease Autonomic Nervous System Diseases Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Movement Disorders Multiple System Atrophy Multiple System Atrophy, Parkinson Variant Nervous System Diseases Neurodegenerative Diseases Parkinson Disease Parkinsonian Disorders Primary Dysautonomias Synucleinopathies

Age range

35 year–75 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Zespol Poradni Specjalistycznych - Botaniczna 3 Poradnia Neurologiczna Szpital Uniwersytecki w Krakowie, Krakow, Lesser Poland Voivodeship, Poland

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

This is an observational Study.

Other

This is an observational study.

Primary outcomes

  1. Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)

    Time frame: 18 months

    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.

  2. MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)

    Time frame: 18 Months

    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.

Secondary outcomes

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

    Time frame: 18 months

    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.

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

    Time frame: 18 months

    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.

Sponsors and collaborators

Lead sponsor

AskBio Inc

Industry

Registry information

Official study title

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

Important dates

Study start
2022
Primary completion
2025
Study completion
2025
First posted
Jan 26, 2023
Registry last updated
Jul 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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