Noninterventional Study Evaluating Parkinson's Disease Diary Use

NCT ID: NCT05363046

Last Updated: 2024-07-31

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Total Enrollment: 194 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2022-07-29
• Study Completion Date: 2026-10-31

Brief Summary

This study aims to evaluate the impact of the frequency of assessments on the variability over time, reliability, and compliance for the Parkinson's disease (PD) diary in patients with PD in whom medications do not provide adequate control of symptoms.

Detailed Description

This is a global, multi-center, noninterventional study of patients with PD aged ≥39 to ≤70 years under standard-of-care treatment that will enroll a minimum of approximately 150 participants and up to 400 participants. Participants will be assigned (1:1) to complete the PD diary either on 3 consecutive days in 1 week (Group A) or 2 consecutive days in each of 2 consecutive weeks (Group B) for a given study visit. During the study, data are collected on motor function, quality of life, and use of PD medications at Baseline and at 3, 6, 12, 18, and 24 months.

Conditions

Parkinson's Disease

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Group A

Participants will complete the PD diary on 3 consecutive days in 1 week.

No interventions assigned to this group

Group B

Participants will complete the PD diary on 2 consecutive days in each of 2 consecutive weeks.

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

• ≥39 to ≤70 years of age at signing of informed consent
• Diagnosis of clinically established PD as defined by the Movement Disorder Society (MDS) Clinical Diagnostic Criteria for PD
• Marked levodopa responsiveness at screening per investigator's judgment (eg, an estimated ≥30% improvement of MDS-UPDRS Part III score in the off-medication versus on-medication state)
• A minimum of 3 years and a maximum of 10 years from time of PD diagnosis to the date of screening
• Receiving optimized and stable PD medical therapy for ≥1 month prior to screening or demonstrated intolerance to PD medications per investigator's judgment in agreement with the medical monitor
• ≥2 hours of average daily OFF-time assessed within 3 months of screening by PD diary or per investigator's judgment
• Hoehn and Yahr Stage of 1 to 3 while on PD medication assessed within 3 months of screening or at screening
• Normal cognition as determined by the investigator after review of relevant testing (eg, Montreal Cognitive Assessment score of ≥26, or ≥22 if no significant cognitive impairment as determined by neuropsychological testing)

Exclusion Criteria

• PD with risk of recurrent falls or only tremor-based symptoms
• Diagnosis of primary mitochondrial disorder, epilepsy, stroke, multiple sclerosis, or clinical features suggestive of a neurodegenerative disease other than PD such as Alzheimer's disease
• Any available evidence inconsistent with dopamine deficiency (eg, 18F-DOPA positron emission tomography [PET] or dopamine transporter single-photon emission computed tomography [DAT-SPECT] imaging if performed)
• Moderately severe dyskinesia per investigator's judgment
• Receiving dopamine receptor-blocking agents, including typical neuroleptics, prochlorperazine, and metoclopramide at the time of screening or within 3 months prior to screening
• Treatment with intrajejunal or subcutaneous infusion therapies for PD within 2 months of screening
• History of gene therapy or cell therapy
• Prior surgical or radiation therapy to the brain, including deep brain stimulation and lesion therapy, or prior history of intradural spinal cord surgery
• Receipt of another investigational therapy or device within 2 years of screening unless approved by the medical monitor

• Minimum Eligible Age: 39 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

BlueRock Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Mayo Clinic Neurology

Scottsdale, Arizona, United States

David Geffen School of Medicine University of California Los Angeles

Los Angeles, California, United States

University of California, Irvine

Orange, California, United States

University of Colorado

Aurora, Colorado, United States

Movement Disorders Center of Boca Raton

Boca Raton, Florida, United States

University of Miami Health System

Miami, Florida, United States

Parkinson's Disease and Movement Disorders Center at Northwestern University Feinberg School of Medicine

Chicago, Illinois, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

University of Louisville

Louisville, Kentucky, United States

Tufts Medical Center

Boston, Massachusetts, United States

Mount Sinai West

New York, New York, United States

Weill Cornell Medicine - New York Presbyterian Hospital

New York, New York, United States

University of Rochester Medical Center

Rochester, New York, United States

Cleveland Clinic

Cleveland, Ohio, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Evergreen Health Medical Center

Kirkland, Washington, United States

Toronto Western Hospital

Toronto, Ontario, Canada

Dkd Wiesbaden

Wolfach, Baden-Wurttemberg, Germany

Klinikum der Universität München - Campus Grosshadern

Munich, Bavaria, Germany

Technischen Universitaet Muenchen (TUM)

Munich, Bavaria, Germany

Universitätsklinikum Würzburg - Medizinische Klinik

Würzburg, Bavaria, Germany

Klinik und Poliklinik für Neurologie am Standort Marburg

Marburg, Hesse, Germany

Universitaetsklinikum Duesseldorf AöR

Düsseldorf, , Germany

Universitätsmedizin Rostock

Rostock, , Germany

University Hospital Tuebingen

Tübingen, , Germany

AOU Ospedali Riuniti Umberto I - G.M. Lancisi - G. Salesi

Ancona, , Italy

Azienda Ospedale Università Padova

Padua, , Italy

IRCCS San Raffaele Pisana

Rome, , Italy

AOU OO.RR. San Giovanni di Dio Ruggi d'Aragona

Salerno, , Italy

Hospital San Juan de Dios

Santurtzi, Bilbao, Spain

Hospital de la Santa Creu i Sant Pau

Barcelona, , Spain

Complexo Hospitalario de Pontevedra (CHOP)

Pontevedra, , Spain

Hospital Universitario Virgen del Rocio

Seville, , Spain

Hospital Universitario Y Politécnico La Fe

Valencia, , Spain

Countries

United States; Canada; Germany; Italy; Spain

Other Identifiers

BRT-DA01-NIS-001

Identifier Type: -

Identifier Source: org_study_id