Clinical trial NCT06680830: A Phase 2 Study of NEU-411 in Companion Diagnostic-Positive Participants With Early Parkinson's Disease
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of NEU-411 in Companion Diagnostic-Positive Participants With Early Parkinson's Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Parkinson Disease
Parkinson
Idiopathic Parkinson Disease
Early Parkinson Disease (Early PD)
Parkinson Disease, Idiopathic
Keywords: Early PD
Parkinsons
Parkinsons Disease
Idiopathic Parkinsons Disease
leucine-rich repeat kinase 2
PD
NEULARK
LRRK2
Lead sponsor: Neuron23 Inc.
Sponsor class: INDUSTRY
Collaborators: name: Qiagen Manchester Limited
name: Roche Diagnostic Ltd.
First posted: 2024-11-08
Enrollment: 150
Sex: ALL
Minimum age (years): 50
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Change from baseline in the Roche digital biomarker score using the Roche Parkinson's Disease application (v3.0) compared to placebo
timeFrame: From enrollment to the end of treatment at 52 weeks
measure: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) compared to placebo
timeFrame: From enrollment to the end of study at 54 weeks
Secondary outcomes:
measure: Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Sun City
country: United States
facility: Banner Sun Health Research Institute
state: Arizona
city: Little Rock
country: United States
facility: University of Arkansas
state: Arkansas
city: Fresno
country: United States
facility: Neuro-Pain Medical Center
state: California
city: Irvine
country: United States
facility: University of California, Irvine
state: California
city: Los Angeles
country: United States
facility: University of California, Los Angeles
state: California
city: Palo Alto
country: United States
facility: Parkinson's Research Centers of America - Palo Alto
state: California
city: San Jose
country: United States
facility: Adaptive Research - California Neuro Institute
state: California
city: Aurora
country: United States
facility: University of Colorado - Anschutz Medical Campus
state: Colorado
city: New Haven
country: United States
facility: Invicro
state: Connecticut
city: Boca Raton
country: United States
facility: Parkinson's Disease and Movement Disorders Center of Boca Raton
state: Florida
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Orlando
country: United States
facility: Neurology One
state: Florida
city: Tampa
country: United States
facility: USF Parkinson's & Movement Disorders Clinic
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Chicago
country: United States
facility: Northwestern Medical Group, Department of Neurology
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Lexington
country: United States
facility: University of Kentucky
state: Kentucky
city: Baltimore
country: United States
facility: University of Maryland
state: Maryland
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
city: Farmington Hills
country: United States
facility: Quest Research Institute
state: Michigan
city: Golden Valley
country: United States
facility: Struthers Parkinson Center
state: Minnesota
city: Las Vegas
country: United States
facility: Cleveland Clinic
state: Nevada
city: Lebanon
country: United States
facility: Darthmouth-Hitchcock Medical Center - Parkinson's Center
state: New Hampshire
city: Great Neck
country: United States
facility: Northwell Health
state: New York
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai/Mount Sinai West
state: New York
city: New York
country: United States
facility: Columbia University
state: New York
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina - Chapel Hill
state: North Carolina
city: Raleigh
country: United States
facility: Velocity Clinical Research at Raleigh Neurology
state: North Carolina
city: Columbus
country: United States
facility: The Ohio State University
state: Ohio
city: Tulsa
country: United States
facility: The Movement Disorder Clinic of Oklahoma
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health & Science University (OHSU)
state: Oregon
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: The University of Texas Health Science Center at Houston
state: Texas
city: Round Rock
country: United States
facility: Central Texas Neurology Consultants
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Movement Division
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
city: Kirkland
country: United States
facility: EvergreenHealth
state: Washington
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
affiliation: Neuron23 Inc.
name: Fatta B Nahab, MD, FAAN FANA
role: PRINCIPAL_INVESTIGATOR
References: