Noninterventional Study Evaluating Parkinson's Disease Diary Use

BlueRock Therapeutics

Status

Active, not recruiting

Conditions

Parkinson's Disease

Study type

Observational

Funder types

Industry

Identifiers

NCT05363046

BRT-DA01-NIS-001

Details and patient eligibility

About

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.

Full 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.

Enrollment

194 patients

Sex

All

Ages

39 to 70 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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