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

December 11, 2023 updated by: Asklepios Biopharmaceutical, Inc.

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

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 Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

150

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Poland
      • Katowice, Poland
        • Recruiting
        • Specjalistyczna Praktyka Lekarska, Prof. Grzegorz Opala
        • Contact:
        • Principal Investigator:
          • Grzegorz Opala, M.D.
      • Warszawa, Poland
        • Recruiting
        • Mazowiecki Szpital Bródnowski w Warszawie, Oddział Neurologii
        • Contact:
        • Principal Investigator:
          • Dariusz Koziorowski, M.D.
      • Warszawa, Poland
        • Recruiting
        • Samodzielny Publiczny Szpital Kliniczny im. prof. Witolda Orłowskiego, Oddział Neurologii i Epileptologii
        • Contact:
        • Contact:
        • Principal Investigator:
          • Ursula Fiszer, M.D.
    • Lesser Poland Voivodeship
      • Kraków, Lesser Poland Voivodeship, Poland
        • Recruiting
        • Zespol Poradni Specjalistycznych - Botaniczna 3 Poradnia Neurologiczna Szpital Uniwersytecki w Krakowie
        • Contact:
        • Contact:
        • Principal Investigator:
          • Agnieszka Slowik
  • United States
    • California
      • Irvine, California, United States, 92697
        • Recruiting
        • University of California Irvine
        • Contact:
        • Principal Investigator:
          • Nicolas Phielipp, M.D.
    • Florida
      • Boca Raton, Florida, United States, 33486
        • Recruiting
        • Parkinson's Disease and Movement Disorders Center of Boca Raton
        • Contact:
        • Principal Investigator:
          • Stuart Isaacson, M.D.
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital
        • Contact:
        • Principal Investigator:
          • Todd Herrington, M.D., PhD
    • Michigan
      • Farmington Hills, Michigan, United States, 48334
        • Recruiting
        • Quest Research Institute
        • Principal Investigator:
          • Aaron Ellenbogen, DO, MPH
        • Contact:
          • Study Coordinator
    • Ohio
      • Columbus, Ohio, United States, 43214
        • Recruiting
        • Ohio Health Research Institute
        • Contact:
        • Principal Investigator:
          • Shnehal Patel, M.D.
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • OHSU Parkinson Center & Movement Disorders Program
        • Principal Investigator:
          • Joseph Quinn, M.D.
        • Contact:
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Recruiting
        • Vanderbilt University Medical Center
        • Principal Investigator:
          • David Charles, M.D.
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

35 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Early Parkinson's disease
Other: This is an observational study.
This is an observational study.
Possible or Probable MSA-P
Other: This is an observational study.
This is an observational study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
18 months
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.
18 Months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
18 months
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.
18 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Asklepios Biopharmaceutical, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 29, 2022

Primary Completion (Estimated)

August 1, 2025

Study Completion (Estimated)

October 1, 2025

Study Registration Dates

First Submitted

October 13, 2022

First Submitted That Met QC Criteria

January 17, 2023

First Posted (Actual)

January 26, 2023

Study Record Updates

Last Update Posted (Actual)

December 12, 2023

Last Update Submitted That Met QC Criteria

December 11, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ASK-PD-0-CS-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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