Only 9 spots left

~9 spots leftby Jun 2029

PR001 for Parkinson's Disease

Recruiting in Palo Alto (17 mi)

+9 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Recruiting

Sponsor: Prevail Therapeutics

Must be taking: ERT, SRT

Must not be taking: Ambroxol, Blood thinners

Disqualifiers: CNS disease, Cancer, Diabetes, others

No Placebo Group

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?

This trial is testing a new treatment called LY3884961 for people with moderate to severe Parkinson's disease who have a specific genetic mutation. The treatment is given directly into the brain to see if it can help improve their symptoms. The study will last for several years to monitor safety and effectiveness.

Will I have to stop taking my current medications?

The trial requires that you have been on a stable dose of your current medications for at least 8 weeks before starting the study. If you are taking treatments for Gaucher Disease, you need to be on a stable regimen for at least 3 months before screening. Some medications, like ambroxol and certain blood thinners, must be stopped before the study.

What data supports the effectiveness of the drug PR001 for Parkinson's Disease?

Research suggests that targeting the LRRK2 gene, which is linked to Parkinson's disease, is a promising approach. Studies on LRRK2 inhibitors, similar to PR001, show potential in treating Parkinson's by slowing disease progression.¹ ² ³ ⁴ ⁵

Research Team

Travis B. Lewis, MD, PhD

Principal Investigator

Prevail Therapeutics

Eligibility Criteria

This trial is for adults with Parkinson's Disease who have a specific GBA1 mutation, weigh between 88-242 lbs with a BMI of 18-34, and are in moderate to severe stages (Hoehn and Yahr Stage III-IV). They must be on stable medications for Parkinson's or Gaucher Disease treatments if applicable. Participants need a reliable informant, updated vaccinations, and agree to use effective contraception if necessary. Exclusions include other significant CNS diseases, certain MRI findings, hypersensitivity to study drugs, uncontrolled health conditions like hypertension or diabetes.

Inclusion Criteria

My weight is between 88 and 242 lbs, and my BMI is between 18 and 34.

I have had the pneumococcal and shingles vaccines within the last 10 years.

See 15 more

Exclusion Criteria

Criterion: You have any of the following conditions or diseases that could interfere with the study, pose a safety risk, or make it difficult for you to follow the study procedures: liver problems, unstable autoimmune disease, uncontrolled diabetes, heart issues, uncontrolled high blood pressure, recent history of certain cancers, recent alcohol or drug abuse, current psychiatric diagnosis that may interfere with study procedures, risk of self-harm, spinal diseases, bleeding problems, recent stroke, recent seizures, active or severe infections, severe allergies, history of certain infections, abnormal lab test results, recent participation in another study with disease-modifying effects on PD, recent gene or cell therapy, recent live vaccinations, recent use of certain medications, contraindications or intolerance to imaging methods, contraindications to anesthesia or sedation, positive urine test for drugs of abuse without prescription, frailty or medical condition that may not be in your best interest to participate in the study.

I don't have allergies or reasons I can't use corticosteroids or sirolimus, including recent severe osteoporosis, uncontrolled high blood pressure, diabetes, or cholesterol.

I have a brain condition that is not Parkinson's but could be causing my symptoms.

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Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intracisternal administration of LY3884961 and are evaluated for safety, tolerability, immunogenicity, biomarkers, and clinical efficacy measures

12 months

Follow-up

Participants are monitored for safety and selected biomarker and efficacy measures

48 months

Treatment Details

Interventions

PR001 (Enzyme Replacement Therapy)

Trial OverviewThe PROPEL trial tests the safety of PR001 (LY3884961), an investigational drug administered into the spinal fluid for those with Parkinson's linked to GBA1 mutations. It includes two dosage levels over five years—initially focusing on safety and efficacy within the first year followed by long-term effects monitoring.

Participant Groups

2Treatment groups

Experimental Treatment

Group I: Dose Level 2Experimental Treatment3 Interventions

Group II: Dose Level 1Experimental Treatment3 Interventions

Find a Clinic Near You

Who Is Running the Clinical Trial?

Prevail Therapeutics

Lead Sponsor

Trials

Recruited

190+

Eli Lilly and Company

Industry Sponsor

Trials

2,708

Recruited

3,720,000+

Dr. Daniel Skovronsky

Eli Lilly and Company

Chief Medical Officer since 2018

MD from Harvard Medical School

David A. Ricks

Eli Lilly and Company

Chief Executive Officer since 2017

BSc from Purdue University, MBA from Indiana University

Findings from Research

A new drug has demonstrated potential in delaying the onset of Parkinsonism, which is a significant advancement for patients at risk of developing this condition.

The findings suggest that this drug could improve the quality of life for individuals by postponing the debilitating symptoms associated with Parkinson's disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Delaying the onset of parkinson's disease.[2016]