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

Active, not recruiting

• Conditions: Multiple System Atrophy, Parkinson Variant; Parkinson's Disease; Multiple System Atrophy
• Interventions: Other: This is an observational study.

• Registration Number: NCT05699460
• Lead Sponsor: Asklepios Biopharmaceutical, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2022-09-29
• Study First Submitted: 2022-10-13
• Study First Submitted QC: 2023-01-17
• Study First Posted: 2023-01-26
• Status Verified: 2024-06
• Last Update Submitted: 2024-06-06
• Last Update Posted: 2024-06-07
• Primary Completion: 2025-08
• Study Completion: 2025-10

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 150

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Possible or Probable MSA-P	This is an observational study.	-
Early Parkinson's disease	This is an observational study.	-

Primary Outcome Measures

Name	Time	Method
Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)	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.
MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)	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 Outcome Measures

Name	Time	Method
Change in the quality of life as measured by Parkinson's Disease Questionnaire (PDQ-39)	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.
Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)	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.

Trial Locations

Locations (11)

Samodzielny Publiczny Szpital Kliniczny im. prof. Witolda Orłowskiego, Oddział Neurologii i Epileptologii; 🇵🇱 Warszawa, Poland

Specjalistyczna Praktyka Lekarska, Prof. Grzegorz Opala; 🇵🇱 Katowice, Poland

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

Quest Research Institute; 🇺🇸 Farmington Hills, Michigan, United States

Ohio Health Research Institute; 🇺🇸 Columbus, Ohio, United States

OHSU Parkinson Center & Movement Disorders Program; 🇺🇸 Portland, Oregon, United States

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States

Mazowiecki Szpital Bródnowski w Warszawie, Oddział Neurologii; 🇵🇱 Warszawa, Poland

Zespol Poradni Specjalistycznych - Botaniczna 3 Poradnia Neurologiczna Szpital Uniwersytecki w Krakowie; 🇵🇱 Kraków, Lesser Poland Voivodeship, Poland