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Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)
NCT04127578 · View on ClinicalTrials.gov ↗
Study 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.
Conditions Studied
Interventions
- DRUG Sirolimus
- DRUG Methylprednisolone
- BIOLOGICAL LY3884961
Study Locations (15)
Other
- Shaare Zedek Medical Center, 12 Shmuel Biet Street — Jerusalem
- Hadassah Medical Center, Hadassah Ein Kerem, Dept. of Neurology, P.O. Box 12000 — Jerusalem
- Sheba Medical Center, Tel Hashomer — Ramat Gan
- Tel Aviv Sourasky Medical Center, 6 Weizmann St. — Tel Aviv
Florida
- K2 Medical Research, 101 Southhall Lane, Suite 150 — Maitland
- PPD, 100 West Gore Street, Suite 202 — Orlando
New York
- Mount Sinai Beth Israel, 10 Union Square East, Suite 5H — New York
- Joan and Sanford I. Weill Department of Medicine, 525 E 68th Street — New York
Arizona
- Mayo Clinic, 13400 E. Shea Boulevard — Scottsdale
California
- Esperanza Clinical, 25220 Hancock Avenue — Murrieta
Colorado
- Rocky Mountain Clinical Research - CenExel - PPDS, 701 East Hampden Avenue Suite 510 — Englewood
Illinois
- Northwestern University Feinberg School of Medicine, Dept. of Neurology, Parkinson's Disease & Movement Disorders Center, 710 N. Lake Shore Drive, 11th Floor — Chicago
Minnesota
- Mayo Clinic, 200 First Street SW — Rochester
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 32 participants |
| Start Date | 2020-01-03 |
| Est. Completion | 2030-12-31 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04127578
The ClinicalTrials.gov registry entry for NCT04127578 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 32 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Prevail Therapeutics, which has 5 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Parkinson Disease appearing as the primary indexed condition, and to 3 interventions — of which Sirolimus is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04127578 reports 15 study locations spanning 10 distinct geographic areas — top geographies include Other, Florida, New York. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04127578 about?
NCT04127578 is a clinical study titled "Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)". 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 LY3884...
What is the current status of trial NCT04127578?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 32 participants. The study started on 2020-01-03. Estimated completion is 2030-12-31.
What conditions does trial NCT04127578 study?
This clinical trial studies the following conditions: Parkinson Disease. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04127578?
The interventions under investigation include: Sirolimus (DRUG), Methylprednisolone (DRUG), LY3884961 (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04127578?
This trial is sponsored by Prevail Therapeutics, which has 5 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04127578 being conducted?
This trial has 15 study locations across Arizona, California, Colorado, Florida, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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