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;
. Unstable autoimmune disease; autoimmune disease requiring chronic immunosuppression;
. Poorly controlled/not adequately managed diabetes (Screening glycosylated hemoglobin \[HbA1C\] ≥ 7%);
. 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.

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

Cumulative number of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Timeframe: 5 years

Incidence of procedure or treatment-emergent AEs measured by brain MRI, spine MRI and nerve conduction study (NCS)

Timeframe: 5 years

Treatment emergent immunogenicity of AAV9 in blood

Timeframe: Thru month 24

Change from baseline in immunogenicity of AAV9 in blood

Timeframe: Baseline and Month 24

Treatment emergent immunogenicity of GCase in blood

Timeframe: Thru Month 24

Change from baseline in immunogenicity of GCase in blood

Timeframe: Baseline and Months 24

Treatment emergent immunogenicity of Nfl in blood

Timeframe: Thru Month 24

Trial details

NCT IDNCT04127578

SponsorPrevail Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2030-12-31

View on ClinicalTrials.gov More Parkinson Disease trials

Plain-language summary

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;
. Unstable autoimmune disease; autoimmune disease requiring chronic immunosuppression;
. Poorly controlled/not adequately managed diabetes (Screening glycosylated hemoglobin \[HbA1C\] ≥ 7%);
. 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;

What they're measuring

Cumulative number of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Timeframe: 5 years

Incidence of procedure or treatment-emergent AEs measured by brain MRI, spine MRI and nerve conduction study (NCS)

Timeframe: 5 years

Treatment emergent immunogenicity of AAV9 in blood

Timeframe: Thru month 24

Change from baseline in immunogenicity of AAV9 in blood

Timeframe: Baseline and Month 24

Treatment emergent immunogenicity of GCase in blood

Timeframe: Thru Month 24

Change from baseline in immunogenicity of GCase in blood

Timeframe: Baseline and Months 24

Treatment emergent immunogenicity of Nfl in blood

Timeframe: Thru Month 24