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: Drug: NEU-411; 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 40-80 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.

Study Timeline

• Study First Submitted: 2024-11-05
• Study First Submitted QC: 2024-11-06
• Study First Posted: 2024-11-08
• Study Start: 2025-01-17
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-17
• Last Update Posted: 2025-07-22
• Primary Completion: 2026-09
• Study Completion: 2027-09-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

1. Aged 40-80 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

Group	Intervention	Description
NEU-411	NEU-411	Orally-administered NEU-411
Placebo	Placebo	Orally-administered matching placebo

Primary Outcome Measures

Name	Time	Method
Change from baseline in the Roche digital biomarker score using the Roche Parkinson's Disease application (v3.0) compared to placebo	From enrollment to the end of treatment at 52 weeks
Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) compared to placebo	From enrollment to the end of study at 54 weeks

Secondary Outcome Measures

Name	Time	Method
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)

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

Esperanza Clinical; 🇺🇸 Murrieta, California, United States

Parkinson's Research Centers of America - Palo Alto; 🇺🇸 Palo Alto, 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

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