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

N/AActive Not RecruitingNCT05699460

AskBio Inc150 enrolled

Overview

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 Type

OBSERVATIONAL

Enrollment

150

Conditions

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

Interventions

This is an observational study.OTHER

This is an observational study.

Eligibility

Sex: ALLMin age: 35 YearsMax age: 75 Years

Medical Language ↔ Plain English

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.

Locations (11)

University of California Irvine

Irvine, California, United States

Parkinson's Disease and Movement Disorders Center of Boca Raton

Boca Raton, Florida, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Outcomes

Primary Outcomes

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.

Time frame: 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.

Time frame: 18 Months

Secondary Outcomes

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.

Time frame: 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.

Time frame: 18 months

Data from ClinicalTrials.gov

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