Clinical trial NCT05363046: Noninterventional Study Evaluating Parkinson's Disease Diary Use
Official title: A Multi-center, Noninterventional Study Evaluating Variability, Reliability, and Compliance for the Parkinson's Disease Diary
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Parkinson's Disease
Lead sponsor: BlueRock Therapeutics
Sponsor class: INDUSTRY
First posted: 2022-05-05
Enrollment: 194
Sex: ALL
Minimum age (years): 39
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Parkinson's disease patients whose medications do not provide adequate control of their symptoms.

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.

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

Primary outcomes:
measure: Change in Good ON-time as measured by the PD Diary.
timeFrame: Baseline, 3, 6, 12, 18 and 24 months.
measure: Individual participant variability for ON-time without troublesome dyskinesia (Good ON-time) as measured by the PD diary.
timeFrame: Baseline, 3, 6, 12, 18 and 24 months.
measure: Proportion of valid PD Diaries.
timeFrame: Baseline, 3, 6, 12, 18 and 24 months.
Secondary outcomes:
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Mayo Clinic Neurology
state: Arizona
city: Los Angeles
country: United States
facility: David Geffen School of Medicine University of California Los Angeles
state: California
city: Orange
country: United States
facility: University of California, Irvine
state: California
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: Boca Raton
country: United States
facility: Movement Disorders Center of Boca Raton
state: Florida
city: Miami
country: United States
facility: University of Miami Health System
state: Florida
city: Chicago
country: United States
facility: Parkinson's Disease and Movement Disorders Center at Northwestern University Feinberg School of Medicine
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Louisville
country: United States
facility: University of Louisville
state: Kentucky
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: New York
country: United States
facility: Mount Sinai West
state: New York
city: New York
country: United States
facility: Weill Cornell Medicine - New York Presbyterian Hospital
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Kirkland
country: United States
facility: Evergreen Health Medical Center
state: Washington
city: Toronto
country: Canada
facility: Toronto Western Hospital
state: Ontario
city: Wolfach
country: Germany
facility: Dkd Wiesbaden
state: Baden-Württemberg
city: Munich
country: Germany
facility: Klinikum der Universität München - Campus Grosshadern
state: Bayern
city: Munich
country: Germany
facility: Technischen Universitaet Muenchen (TUM)
state: Bayern
city: Würzburg
country: Germany
facility: Universitätsklinikum Würzburg - Medizinische Klinik
state: Bayern
city: Marburg
country: Germany
facility: Klinik und Poliklinik für Neurologie am Standort Marburg
state: Hessen
city: Düsseldorf
country: Germany
facility: Universitaetsklinikum Duesseldorf AöR
city: Rostock
country: Germany
facility: Universitätsmedizin Rostock
city: Tübingen
country: Germany
facility: University Hospital Tuebingen
city: Ancona
country: Italy
facility: AOU Ospedali Riuniti Umberto I - G.M. Lancisi - G. Salesi
city: Padova
country: Italy
facility: Azienda Ospedale Università Padova
city: Rome
country: Italy
facility: IRCCS San Raffaele Pisana
city: Salerno
country: Italy
facility: AOU OO.RR. San Giovanni di Dio Ruggi d'Aragona
city: Santurtzi
country: Spain
facility: Hospital San Juan de Dios
state: Bilbao
city: Barcelona
country: Spain
facility: Hospital de la Santa Creu i Sant Pau
city: Pontevedra
country: Spain
facility: Complexo Hospitalario de Pontevedra (CHOP)
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
city: Valencia
country: Spain
facility: Hospital Universitario Y Politécnico La Fe
Study officials:
References: