MedPath

A Phase 2 Study of NEU-411 in Companion Diagnostic-Positive Participants With Early Parkinson's Disease

Phase 2
Recruiting
Conditions
Parkinson Disease
Parkinson
Idiopathic Parkinson Disease
Early Parkinson Disease (Early PD)
Parkinson Disease, Idiopathic
Interventions
Other: Placebo
Registration Number
NCT06680830
Lead Sponsor
Neuron23 Inc.
Brief Summary

The goal of this Phase 2 clinical trial is to investigate the efficacy and safety of NEU-411 in men and women aged 50-80 years with early Parkinson's Disease (PD) who have predicted elevations in the activity of the "leucine-rich repeat kinase 2" ("LRRK2" for short) pathway based on their genetic profile. A DNA test will be used to identify the "LRRK2-driven" population with predicted elevation in the LRRK2 pathway.

Participants will:

• Take NEU-411 or placebo every day for 52 weeks

Detailed Description

NEU-411-PD201 is a Phase 2, randomized, placebo-controlled, proof-of-concept study in participants with early Parkinson's Disease (PD) who have LRRK2-driven PD as measured by an investigational companion diagnostic genetic test (CDx). The study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of NEU-411, an orally-administered, potent, selective, bioavailable, highly permeable, brain penetrant, small molecule inhibitor of LRRK2 activity as compared to placebo.

After participants are screened for inclusion in the study, approximately 150 participants will be randomized in a 1:1 allocation ratio to NEU-411 30 mg once per day or placebo for a 52-week treatment period. A safety follow-up visit will occur 2 weeks after the last treatment visit.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
150
Inclusion Criteria
  1. Aged 50-80 years at time of screening, inclusive
  2. Diagnosis of clinically established or clinically probable Parkinson's Disease (PD)
  3. LRRK2-driven PD using the investigational companion diagnostic genetic test (CDx)
  4. Modified Hoehn and Yahr (mH&Y) of 1 to 2.5
Exclusion Criteria
  1. Secondary or atypical parkinsonian syndromes
  2. Uncontrolled diabetes mellitus with hemoglobin A1c (HbA1c) >8%
  3. Other significant medical conditions (as determined by medical history, examination, or clinical investigations at screening)

Additional inclusion and exclusion criteria are outlined in the full study protocol.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
NEU-411NEU-411Orally-administered NEU-411
PlaceboPlaceboOrally-administered matching placebo
Primary Outcome Measures
NameTimeMethod
Change from baseline in the Roche digital biomarker score using the Roche Parkinson's Disease application (v3.0) compared to placeboFrom enrollment to the end of treatment at 52 weeks
Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) compared to placeboFrom enrollment to the end of study at 54 weeks
Secondary Outcome Measures
NameTimeMethod
Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)52 weeks

Change from baseline to Week 52 in motor function/nonmotor as measured by Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)

Trial Locations

Locations (43)

Barrow Neurological Institute

🇺🇸

Phoenix, Arizona, United States

Banner Sun Health Research Institute

🇺🇸

Sun City, Arizona, United States

University of Arkansas

🇺🇸

Little Rock, Arkansas, United States

Neuro-Pain Medical Center

🇺🇸

Fresno, California, United States

University of California, Irvine

🇺🇸

Irvine, California, United States

University of California, Los Angeles

🇺🇸

Los Angeles, California, United States

Parkinson's Research Centers of America - Palo Alto

🇺🇸

Palo Alto, California, United States

Adaptive Research - California Neuro Institute

🇺🇸

San Jose, California, United States

University of Colorado - Anschutz Medical Campus

🇺🇸

Aurora, Colorado, United States

Invicro

🇺🇸

New Haven, Connecticut, United States

Parkinson's Disease and Movement Disorders Center of Boca Raton

🇺🇸

Boca Raton, Florida, United States

University of Florida

🇺🇸

Gainesville, Florida, United States

Neurology One

🇺🇸

Orlando, Florida, United States

USF Parkinson's & Movement Disorders Clinic

🇺🇸

Tampa, Florida, United States

Emory University

🇺🇸

Atlanta, Georgia, United States

Augusta University

🇺🇸

Augusta, Georgia, United States

Northwestern Medical Group, Department of Neurology

🇺🇸

Chicago, Illinois, United States

University of Kansas Medical Center

🇺🇸

Kansas City, Kansas, United States

University of Kentucky

🇺🇸

Lexington, Kentucky, United States

University of Maryland

🇺🇸

Baltimore, Maryland, United States

Beth Israel Deaconess Medical Center

🇺🇸

Boston, Massachusetts, United States

Quest Research Institute

🇺🇸

Farmington Hills, Michigan, United States

Struthers Parkinson Center

🇺🇸

Golden Valley, Minnesota, United States

Cleveland Clinic

🇺🇸

Las Vegas, Nevada, United States

Darthmouth-Hitchcock Medical Center - Parkinson's Center

🇺🇸

Lebanon, New Hampshire, United States

Northwell Health

🇺🇸

Great Neck, New York, United States

Icahn School of Medicine at Mount Sinai/Mount Sinai West

🇺🇸

New York, New York, United States

Columbia University

🇺🇸

New York, New York, United States

University of Rochester

🇺🇸

Rochester, New York, United States

University of North Carolina - Chapel Hill

🇺🇸

Chapel Hill, North Carolina, United States

Velocity Clinical Research at Raleigh Neurology

🇺🇸

Raleigh, North Carolina, United States

The Ohio State University

🇺🇸

Columbus, Ohio, United States

The Movement Disorder Clinic of Oklahoma

🇺🇸

Tulsa, Oklahoma, United States

Oregon Health & Science University (OHSU)

🇺🇸

Portland, Oregon, United States

Medical University of South Carolina

🇺🇸

Charleston, South Carolina, United States

Vanderbilt University

🇺🇸

Nashville, Tennessee, United States

Baylor College of Medicine

🇺🇸

Houston, Texas, United States

The University of Texas Health Science Center at Houston

🇺🇸

Houston, Texas, United States

Central Texas Neurology Consultants

🇺🇸

Round Rock, Texas, United States

University of Utah Movement Division

🇺🇸

Salt Lake City, Utah, United States

University of Virginia

🇺🇸

Charlottesville, Virginia, United States

EvergreenHealth

🇺🇸

Kirkland, Washington, United States

Medical College of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

© Copyright 2025. All Rights Reserved by MedPath