Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least… (NCT04127578) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)
United States, Israel32 participantsStarted 2020-01-03
Plain-language summary
Study J3Z-MC-OJAA is a Phase 1/2a, multicenter, open-label, ascending dose, first in-human study that will evaluate the safety of intracisternal LY3884961 administration in patients with moderate to severe Parkinson's disease with at least 1 pathogenic GBA1 mutation. Two dose level cohorts of LY3884961 are planned (Dose Level 1 and Dose Level 2). The duration of the study is 5 years. During the first year, patients will be evaluated for the effect of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and clinical efficacy measures. Patients will continue to be followed for an additional 4 years to continue to monitor safety as well as selected biomarker and efficacy measures.
Who can participate
Age range
35 Years – 80 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.
Exclusion criteria
. Evidence of clinically significant liver pathology;
. History of unstable angina, myocardial infarction, chronic heart failure (New York Heart Association Class III or IV), or clinically significant conduction abnormalities (e.g., unstable atrial fibrillation) within 1 year prior to Screening;
. Clinically significant 12-lead electrocardiogram (ECG) abnormalities at Screening, as determined by the Investigator;
. Uncontrolled hypertension;
. History of cancer, including B-cell cancers, within 5 years of Screening with the exception of fully excised non-melanoma skin cancers, non-metastatic prostate cancer, and full treated ductal carcinoma in situ, provided it has been stable for at least 6 months;
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 trial uses a gene therapy called PR001 delivered directly into the spinal fluid — what does that delivery process involve, and what are the main risks I should understand given that this is still in Phase 1/2a where safety is the primary focus?
2Since the trial is specifically for Parkinson's patients who carry at least one GBA1 mutation, would you recommend I get genetic testing first to find out if I have this mutation, and how would that result affect my treatment options overall?
3The trial is actively monitoring immune reactions to the AAV9 virus used to deliver the gene therapy — what does it mean for my safety if my immune system reacts strongly to it, and how would that be managed?
4The trial is no longer enrolling new patients — does that affect my options, and is there anything about the safety or early results from this study that might be relevant to the treatment decisions we're making for me now?
5Given that this is an early-phase gene therapy trial focused on safety rather than proven benefit, how would you weigh this approach against standard Parkinson's treatments, especially since the gene therapy effect is likely irreversible once given?
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
Cumulative number of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timeframe: 5 years
2
Incidence of procedure or treatment-emergent AEs measured by brain MRI, spine MRI and nerve conduction study (NCS)
Timeframe: 5 years
3
Treatment emergent immunogenicity of AAV9 in blood
Timeframe: Thru month 24
4
Change from baseline in immunogenicity of AAV9 in blood
Timeframe: Baseline and Month 24
5
Treatment emergent immunogenicity of GCase in blood
Timeframe: Thru Month 24
6
Change from baseline in immunogenicity of GCase in blood