A Phase 2 Study and Open-Label Extension of NEU-411 in Companion Diagnostic-Positive Participants With Early Parkinson's Disease
Purpose
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.
Conditions
- Parkinson Disease
- Parkinson
- Idiopathic Parkinson Disease
- Early Parkinson Disease (Early PD)
- Parkinson Disease, Idiopathic
Eligibility
- Eligible Ages
- Between 40 Years and 80 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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
- 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 for the RCP and OLE are outlined in the full study protocol.
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Masking Description
- The Sponsor is also masked.
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental NEU-411 |
Orally-administered NEU-411 |
|
|
Placebo Comparator Placebo |
Orally-administered matching placebo |
|
Recruiting Locations
UK Center for Clinical and Translational Science and nearby locations
Lexington 4297983, Kentucky 6254925 40536
More Details
- NCT ID
- NCT06680830
- Status
- Recruiting
- Sponsor
- Neuron23 Inc.
Detailed Description
NEU-411-PD201 is a Phase 2, randomized, placebo-controlled, proof-of-concept study and open-label extension (OLE) 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 randomized, placebo-controlled portion of the study, approximately 150 participants will be randomized in a 1:1 allocation to NEU-411 30 mg once per day or placebo for a 52-week treatment period with a safety follow-up visit within 2 weeks after the last treatment visit. Eligible participants may enroll in the OLE and receive treatment for an additional 26 weeks.