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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) | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGPHASE1/PHASE2

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

A Phase 1/2a Open-Label Ascending Dose Study to Evaluate the Safety and Effects of LY3884961 in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

NCT04127578•Prevail Therapeutics

View on ClinicalTrials.gov

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.

Eligibility

Age

35 - 80 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Body weight range of ≥40 kg (88 lbs) to ≤110 kg (242 lbs) and a body mass index (BMI) of 18 to 34 kg/m2.
•Diagnosis of Parkinson's Disease (PD) per UK Parkinson's Disease Society Brain Bank Clinical Diagnostic Criteria.
•Hoehn and Yahr Stage III-IV (as determined in Practically Defined OFF state).
•Stable use of background medications at least 8 weeks prior to investigational product (IP) administration, including but not limited to those used for treatment of PD. Gaucher Disease-PD patients receiving GD treatments should be on a stable regimen of their ERT or substrate replacement therapy (SRT) medication for at least 3 months prior to screening.
•At least 1 pathogenic GBA1 mutation confirmed by the central laboratory
•Negative screening test for Mycobacterium tuberculosis (MTB), documented negative MTB test within 1 year prior to Screening, or clearance by an infectious disease specialist.
•Patient and/or patient's legally authorized representative (LAR) has the ability to understand the purpose and risks of the study and provide written informed consent and authorization to use protected health information in accordance with national and local privacy regulations.
•Patient has a reliable study partner/informant (e.g., family member, friend) willing and able to participate in the study as a source of information on the patient's health status and cognitive and functional abilities (including providing input into the rating scales). The study partner should have regular contact with the patient (in person or via phone/video communication). The study partner must sign a separate partner informed consent form (ICF) indicating that she/he understands the study requirements and is willing to participate and attend study visits requiring study partner input.
•Women of nonchildbearing potential must be either surgically sterile or postmenopausal. Men and women of childbearing potential must use a highly effective method of contraception consistently and correctly for the duration of the study including the long-term follow-up. Individuals in an exclusively same sex relationship (as their preferred and usual lifestyle) are not required to use contraception.
•Men must agree to abstain from sperm donation for the duration of the study, including long-term follow-up.
+6 more criteria

Exclusion Criteria

•Diagnosis of a significant CNS disease other than Parkinson's Disease (PD) that may be a cause for the patient's PD symptoms or may confound study objectives.
•MoCA (Montreal Cognitive Assessment) score of \<14
•Spinal, cervical, or brain MRI/magnetic resonance angiography (MRA) indicating clinically significant abnormality, including evidence of prior hemorrhage, infarct \>1 cm3 or \>3 lacunar infarcts, or a structural abnormality deemed a contraindication to intracisternal injection.
•Hypersensitivity or contraindications to corticosteroid and/or, sirolimus use (including but not limited to osteoporosis with vertebral fractures within 1 year prior to Screening, uncontrolled hypertension, poorly controlled diabetes, uncontrolled hyperlipidemia or hypercholesterolemia as per Investigator assessment.
•Concomitant disease or condition within 6 months of Screening that could interfere with, or treatment of which might interfere with, the conduct of the study or that would, in the opinion of the Investigator, pose an unacceptable safety risk to the patient or interfere with the patient's ability to comply with study procedures; including, but not limited to the following:
•1. Evidence of clinically significant liver pathology;
•2. Unstable autoimmune disease; autoimmune disease requiring chronic immunosuppression;
•3. Poorly controlled/not adequately managed diabetes (Screening glycosylated hemoglobin \[HbA1C\] ≥ 7%);
•4. 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;
•5. Clinically significant 12-lead electrocardiogram (ECG) abnormalities at Screening, as determined by the Investigator;
+22 more criteria

Locations (15)

Mayo Clinic, 13400 E. Shea Boulevard

Scottsdale, Arizona, United States

Esperanza Clinical, 25220 Hancock Avenue

Murrieta, California, United States

Rocky Mountain Clinical Research - CenExel - PPDS, 701 East Hampden Avenue Suite 510

Englewood, Colorado, United States

K2 Medical Research, 101 Southhall Lane, Suite 150

Maitland, Florida, United States

PPD, 100 West Gore Street, Suite 202

Orlando, Florida, United States

Northwestern University Feinberg School of Medicine, Dept. of Neurology, Parkinson's Disease & Movement Disorders Center, 710 N. Lake Shore Drive, 11th Floor

Chicago, Illinois, United States

Mayo Clinic, 200 First Street SW

Rochester, Minnesota, United States

Mount Sinai Beth Israel, 10 Union Square East, Suite 5H

New York, New York, United States

Joan and Sanford I. Weill Department of Medicine, 525 E 68th Street

New York, New York, United States

Hospital of the University of Pennsylvania, 330 S. 9th Street

Philadelphia, Pennsylvania, United States

Key Dates

Start Date

January 3, 2020

Primary Completion Date

December 31, 2030

Completion Date

December 31, 2030

Last Updated

March 13, 2026

Enrollment

ESTIMATED participants

Conditions

Parkinson Disease

Interventions

LY3884961

BIOLOGICAL

Methylprednisolone

DRUG

Sirolimus

DRUG

Characterizing the Pathophysiological Role of the Pallido-thalamocortical Motor Pathway in Parkinson's Disease.

NCT06692920

Parkinson Disease

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RECRUITINGPHASE1

[18F]F-DOPA Imaging in Patients With Autonomic Failure

NCT04246437

Autonomic FailurePure Autonomic Failure+3 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 13, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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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)

Inclusion Criteria

Exclusion Criteria

Characterizing the Pathophysiological Role of the Pallido-thalamocortical Motor Pathway in Parkinson's Disease.

[18F]F-DOPA Imaging in Patients With Autonomic Failure

Montelukast in Parkinson Disease

Exercise for Cognitive Excellence in Parkinson's Disease

The Effect of Functional Rotational Exercises on Fall Risk and Mobility in Parkinson's Disease Patients

SAD Study in Patients With Parkinson's Disease and Motor Fluctuations