GDNF Gene Therapy for Multiple System Atrophy (NCT04680065) | Clinical Trial Compass
Active — Not RecruitingPhase 1
GDNF Gene Therapy for Multiple System Atrophy
United States9 participantsStarted 2023-10-03
Plain-language summary
The objective of this randomized, double-blinded, placebo-controlled Phase 1 investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a possible or probable diagnosis of Multiple System Atrophy.
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:
* Male and female 35-75 years of age (inclusive)
* Clinical diagnosis of MSA, parkinsonian type with symptoms onset sporadic, progressive and \> 30 years of age
* Less than 5 years from MSA parkinsonian diagnosis with expected survival more than 3 years
* Stable anti-parkinsonian medication regimen
* Ability to walk a distance of 25 feet with or without an assistive device
Exclusion Criteria:
* Presence of idiopathic Parkinson's disease (PD) or any PD-related mutation or other neurological diseases
* Presence of dementia, psychosis, substance abuse or poorly controlled depression
* Prior brain surgery (i.e., deep brain stimulator implantation) or other brain imaging abnormalities
* History of cancer or poorly controlled medical conditions that would increase surgical risk
* Received investigational agent within 12 weeks
* Inability to tolerate laying flat in an MRI and/or allergy to gadolinium
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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
The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations