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Noninterventional Study Evaluating Parkinson's Disease Diary Use

Sponsor
BlueRock Therapeutics (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05363046
Collaborator
(none)
500
Enrollment
1
Location
50.1
Anticipated Duration (Months)
10
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase

    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 approximately up to 500 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.

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    500 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    A Multi-center, Noninterventional Study Evaluating Variability, Reliability, and Compliance for the Parkinson's Disease Diary
    Actual Study Start Date :
    Jul 29, 2022
    Anticipated Primary Completion Date :
    Oct 1, 2025
    Anticipated Study Completion Date :
    Oct 1, 2026

    Arms and Interventions

    Arm Intervention/Treatment
    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.

    Outcome Measures

    Primary Outcome Measures

    1. Change in Good ON-time as measured by the PD Diary. [Baseline, 3, 6, 12, 18 and 24 months.]

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

    2. Individual participant variability for ON-time without troublesome dyskinesia (Good ON-time) as measured by the PD diary. [Baseline, 3, 6, 12, 18 and 24 months.]

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

    3. Proportion of valid PD Diaries. [Baseline, 3, 6, 12, 18 and 24 months.]

      Proportion of valid PD Diaries.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    39 Years to 70 Years
    Sexes Eligible for Study:
    All

    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 18 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

    • ≥3 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 PD therapy with deep brain stimulation, lesion therapy, gene therapy, or cell therapy

    • Prior surgical or radiation therapy to the brain or spinal cord

    • Receipt of another investigational therapy or device within 2 years of screening unless approved by the medical monitor

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Movement Disorders Center of Boca Raton Boca Raton Florida United States 33486

    Sponsors and Collaborators

    • BlueRock Therapeutics

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    BlueRock Therapeutics
    ClinicalTrials.gov Identifier:
    NCT05363046
    Other Study ID Numbers:
    • BRT-DA01-NIS-001
    First Posted:
    May 5, 2022
    Last Update Posted:
    Aug 17, 2022
    Last Verified:
    Aug 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Parkinson Disease

    Study Results

    No Results Posted as of Aug 17, 2022