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

Parkinson's Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05699460
• Other study ID #: ASK-PD-0-CS-001
• Status: Recruiting
• Start date: September 29, 2022
• Est. completion date: October 2025

Study information

• Verified date: December 2023
• Source: Asklepios Biopharmaceutical, Inc.
• Contact: Amber Van Laar, M.D.
• Phone: 336-265-2790
• Email: askfirst[@]askbio.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: October 2025
• Est. primary completion date: August 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 35 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Atrophy
• Multiple System Atrophy
• Parkinson Disease
• Parkinson's Disease
• Shy-Drager Syndrome

Intervention

Other:
• This is an observational study.
This is an observational study.

Locations

Country	Name	City	State
Poland	Specjalistyczna Praktyka Lekarska, Prof. Grzegorz Opala	Katowice
Poland	Zespol Poradni Specjalistycznych - Botaniczna 3 Poradnia Neurologiczna Szpital Uniwersytecki w Krakowie	Kraków	Lesser Poland Voivodeship
Poland	Mazowiecki Szpital Bródnowski w Warszawie, Oddzial Neurologii	Warszawa
Poland	Samodzielny Publiczny Szpital Kliniczny im. prof. Witolda Orlowskiego, Oddzial Neurologii i Epileptologii	Warszawa
United States	Parkinson's Disease and Movement Disorders Center of Boca Raton	Boca Raton	Florida
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Ohio Health Research Institute	Columbus	Ohio
United States	Quest Research Institute	Farmington Hills	Michigan
United States	University of California Irvine	Irvine	California
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	OHSU Parkinson Center & Movement Disorders Program	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Asklepios Biopharmaceutical, Inc.

Countries where clinical trial is conducted

United States,  Poland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	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
Secondary	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