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

Sponsor

Asklepios Biopharmaceutical, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05699460

Collaborator

(none)

150

Enrollment

Locations

36.1

Anticipated Duration (Months)

Patients Per Site

0.8

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Parkinson's Disease Multiple System Atrophy, Parkinson Variant Multiple System Atrophy	Other: This is an observational study.

Study Design

Study Type:

Observational

Anticipated Enrollment :

150 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

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

Actual Study Start Date :

Sep 29, 2022

Anticipated Primary Completion Date :

Aug 1, 2025

Anticipated Study Completion Date :

Oct 1, 2025

Arms and Interventions

Arm	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.

Arm

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.

Outcome Measures

Primary Outcome Measures

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

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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

35 Years to 75 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Parkinson's Disease and Movement Disorders Center of Boca Raton	Boca Raton	Florida	United States	33486
2	Quest Research Institute	Farmington Hills	Michigan	United States	48334
3	OHSU Parkinson Center & Movement Disorders Program	Portland	Oregon	United States	97239
4	Specjalistyczna Praktyka Lekarska, Prof. Grzegorz Opala	Katowice		Poland
5	Samodzielny Publiczny Szpital Kliniczny im. prof. Witolda Orłowskiego Oddział Neurologii i Epileptologii	Warszawa		Poland