Early Parkinson Disease (Early PD), Idiopathic Parkinson Disease, Parkinson, Parkinson Disease, Parkinson Disease, Idiopathic
Conditions
Keywords
Early PD, Parkinsons, Parkinsons Disease, Idiopathic Parkinsons Disease, leucine-rich repeat kinase 2, PD, NEULARK, LRRK2, PARK8, de novo Parkinsons disease, Parkinson's disease
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.
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.
Interventions
DRUGNEU-411
NEU-411, a potent, selective, orally bioavailable, highly permeable, brain penetrant, small molecule inhibitor of LRRK2 activity
OTHERPlacebo
Orally-administered matched placebo
Sponsors
Neuron23 Inc.
Qiagen Manchester Limited
Roche Diagnostic Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
The Sponsor is also masked.
Eligibility
Sex/Gender
ALL
Age
40 Years to 80 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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) |
Countries
Israel, Italy, Poland, Spain, United Kingdom, United States
Contacts
CONTACTFatta B Nahab, MD, FAAN, FANA
PRINCIPAL_INVESTIGATORFatta B Nahab, MD, FAAN FANA
Neuron23 Inc.
Outcome results
None listed