A Phase 2 Study and Open-Label Extension of NEU-411 in Companion Diagnostic-Positive Participants With Early Parkinson's Disease

Официальное название

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)

Обзор

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.

Подробное описание

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.

Вмешательства

• Препарат NEU-411
  NEU-411, a potent, selective, orally bioavailable, highly permeable, brain penetrant, small molecule inhibitor of LRRK2 activity
• Другое Placebo
  Orally-administered matched placebo

Первичные конечные точки

• Change from baseline in the Roche digital biomarker score using the Roche Parkinson's Disease application (v3.0) compared to placebo [Срок оценки: 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 [Срок оценки: From enrollment to the end of study at 54 weeks]

Вторичные конечные точки (1)

• Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS) [Срок оценки: 52 weeks]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 45 центров

• Banner Sun Health Research Institute — Sun City
• University of Arkansas — Little Rock
• Neuro-Pain Medical Center — Fresno
• University of California, Irvine — Irvine
• University of California, Los Angeles — Los Angeles
• Esperanza Clinical — Murrieta
• Parkinson's Research Centers of America - Palo Alto — Palo Alto
• Sutter Neuroscience Institute — Sacramento
• … и ещё 37 центров

Испания · 7 центров

• Policlinica Gipuzkoa - Centro de Investigacion Parkinson (CIP) — San Sebastián
• Instituto de Investigacion Sanitaria Biocruces Bizkaia - Hospital Universitario Cruces — Barakaldo
• Hospital Universitari Vall d'Hebron — Barcelona
• Hospital Clinic de Barcelona (Hospital Clinic i Provincial) - Barnaclinic S.A. — Barcelona
• Universidad Autonoma de Madrid (UAM) - Hospital Universitario de La Princesa — Madrid
• Hospital Universitario HM Puerta del Sur — Móstoles
• Hospital Universitario Virgen del Rocio (HUVR) - Instituto de Biomedicina de Sevilla (IBIS — Seville

Италия · 6 центров

• IRCCS Ospedale San Raffaele (HSR) - Dipartimento Di Neurologia — Milan
• Universita Degli Studi Della Campania "Luigi Vanvitelli" - Azienda Ospedaliera Universitar — Naples
• Universita Degli Studi Di Padova - Azienda Ospedaliera Di Padova - Clinica Neurologica — Padua
• Azienda Ospedaliero Universitaria Pisana - Stabilimento Ospedaliero Di Santa Chiara — Pisa
• Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - San Raffaele Pisana — Rome
• Fondazione Policilinico Universitario A Gemelli IRCCS — Rome

Израиль · 4 центра

• Rambam Medical Center — Haifa
• Shaare Zedek Medical Center — Jerusalem
• Sheba Medical Center — Ramat Gan
• Tel Aviv Medical Center — Tel Aviv

Польша · 4 центра

• Centrum Medyczne Neuromed — Bydgoszcz
• NZOZ Wielospecjalistyczna Poradnia Lekarska Synapsis Lech Szczechowski — Katowice
• Pro Life Medica Sp. z o. o. ETA Lublin — Lublin
• Insula Badania Sp. Z o.o. — Warsaw

Великобритания · 4 центра

• St George's Hospital, University of London — London
• University College London Hospitals NHS Foundation Trust — London
• Clinical Aging Research Unit, Newcastle University — Newcastle upon Tyne
• Ninewells Hospital and Medical School - NHS Tayside — Dundee

Идентификаторы

NCT: NCT06680830 · NEU-411-PD201

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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