A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study and Open-Label Extension to Evaluate the Safety and Efficacy of NEU-411 in Companion Diagnostic-Positive Participants With Early Parkinson's Disease (NEULARK)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 150
- 试验地点
- 114
- 主要终点
- Change from baseline in the Roche digital biomarker score using the Roche Parkinson's Disease application (v3.0) compared to placebo
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
The Sponsor is also masked.
入排标准
- 年龄范围
- 40 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 40-80 years at time of screening, inclusive
- •Diagnosis of clinically established or clinically probable Parkinson's Disease (PD)
- •LRRK2-driven PD using the investigational companion diagnostic genetic test (CDx)
- •Modified Hoehn and Yahr (mH&Y) of 1 to 2.5
排除标准
- •Secondary or atypical parkinsonian syndromes
- •Uncontrolled diabetes mellitus with hemoglobin A1c (HbA1c) >8%
- •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.
研究组 & 干预措施
Placebo
Orally-administered matching placebo
干预措施: Placebo (Other)
NEU-411
Orally-administered NEU-411
干预措施: NEU-411 (Drug)
结局指标
主要结局
Change from baseline in the Roche digital biomarker score using the Roche Parkinson's Disease application (v3.0) compared to placebo
时间窗: 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
时间窗: From enrollment to the end of study at 54 weeks
次要结局
- Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)(52 weeks)