Noninterventional Study Evaluating Parkinson's Disease Diary Use

NCT05363046 | Active Not Recruiting

• 1 other identifier: BRT-DA01-NIS-001
• Study Type: observational
• Target: 194
• Locations: 5 countries
• Sites: 34

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.

Conditions

Parkinson's Disease

Outcome Measures

Primary Outcomes (3)

• Change in Good ON-time as measured by the PD Diary.

  Standard deviation of change in ON-time without troublesome dyskinesia (Good ON-time) as measured by the PD diary.

  Baseline, 3, 6, 12, 18 and 24 months.

• Individual participant variability for ON-time without troublesome dyskinesia (Good ON-time) as measured by the PD diary.

  Within-subject coefficient of variation for ON-time without troublesome dyskinesia (Good ON-time) as measured by the PD diary.

  Baseline, 3, 6, 12, 18 and 24 months.

• Proportion of valid PD Diaries.

  Proportion of valid PD Diaries.

  Baseline, 3, 6, 12, 18 and 24 months.

Study Arms (2)

Group A

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

Group B

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

Eligibility Criteria

• Age: 39 Years - 70 Years
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Parkinson's disease patients whose medications do not provide adequate control of their symptoms.

You may qualify if:

• ≥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)

You may not qualify if:

• 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

Sponsors & Collaborators

• BlueRock Therapeutics: lead

Study Sites (34)

Mayo Clinic Neurology

Scottsdale, Arizona, 85259, United States

Location

David Geffen School of Medicine University of California Los Angeles

Los Angeles, California, 90095, United States

Location

University of California, Irvine

Orange, California, 92868, United States

Location

University of Colorado

Aurora, Colorado, 80045, United States

Location

Movement Disorders Center of Boca Raton

Boca Raton, Florida, 33486, United States

Location

University of Miami Health System

Miami, Florida, 33136, United States

Location

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

Chicago, Illinois, 60611, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66103, United States

Location

University of Louisville

Louisville, Kentucky, 40202, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111, United States

Location

Mount Sinai West

New York, New York, 10019, United States

Location

Weill Cornell Medicine - New York Presbyterian Hospital

New York, New York, 10021, United States

Location

University of Rochester Medical Center

Rochester, New York, 14618, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Evergreen Health Medical Center

Kirkland, Washington, 98034, United States

Location

Toronto Western Hospital

Toronto, Ontario, M5T 2S8, Canada

Location

Dkd Wiesbaden

Wolfach, Baden-Wurttemberg, 77709, Germany

Location

Klinikum der Universität München - Campus Grosshadern

Munich, Bavaria, 81377, Germany

Location

Technischen Universitaet Muenchen (TUM)

Munich, Bavaria, 81675, Germany

Location

Universitätsklinikum Würzburg - Medizinische Klinik

Würzburg, Bavaria, 97080, Germany

Location

Klinik und Poliklinik für Neurologie am Standort Marburg

Marburg, Hesse, 35043, Germany

Location

Universitaetsklinikum Duesseldorf AöR

Düsseldorf, 40225, Germany

Location

Universitätsmedizin Rostock

Rostock, 18147, Germany

Location

University Hospital Tuebingen

Tübingen, 72076, Germany

Location

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

Ancona, 60126, Italy

Location

Azienda Ospedale Università Padova

Padua, 35128, Italy

Location

IRCCS San Raffaele Pisana

Rome, 00163, Italy

Location

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

Salerno, 84131, Italy

Location

Hospital San Juan de Dios

Santurtzi, Bilbao, 48980, Spain

Location

Hospital de la Santa Creu i Sant Pau

Barcelona, 08041, Spain

Location

Complexo Hospitalario de Pontevedra (CHOP)

Pontevedra, 36071, Spain

Location

Hospital Universitario Virgen del Rocio

Seville, 41013, Spain

Location

Hospital Universitario Y Politécnico La Fe

Valencia, 46026, Spain

Location

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 3, 2022
• First Posted: May 5, 2022
• Study Start: July 29, 2022
• Primary Completion: October 1, 2025
• Study Completion (Estimated): October 1, 2026
• Last Updated: July 31, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (16); DE (8); IT (4); CA (1); ES (5)