Pre-Gene Therapy Study in Parkinson's Disease and Multiple System Atrophy (NCT05699460) | Clinical Trial Compass
CompletedNot Applicable
Pre-Gene Therapy Study in Parkinson's Disease and Multiple System Atrophy
United States, Poland73 participantsStarted 2022-09-29
Plain-language 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.
Who can participate
Age range
35 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This study has already been completed — would it be possible to see its results, and do those findings about motor symptoms measured by MDS-UPDRS or UMSARS tell us anything relevant to my own situation?
2Since this was described as a 'pre-gene therapy' study, does that mean it was designed to gather baseline data before a future gene therapy trial, and if so, is there an active follow-up gene therapy trial I might be worth discussing as a next step?
3The trial enrolled both Parkinson's Disease and Multiple System Atrophy patients — given my specific diagnosis, how applicable would the findings from this study be to my case, especially since MSA and PD can progress quite differently?
4Because this trial is listed as Phase NA, which sometimes means it was observational rather than testing a treatment, can you help me understand whether participants received any experimental intervention, or whether this was purely a data-collection study to prepare for future trials?
5Are there currently open or upcoming gene therapy trials for Parkinson's or MSA that this completed study was building toward, and would it be worth discussing whether I might be a candidate for one of those next-stage studies?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Timeframe: 18 months
2
MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)