Noninterventional Study Evaluating Parkinson's Disease Diary Use

• ClinicalTrials.gov Identifier: NCT05363046
• Recruitment Status: Recruiting
• First Posted: May 5, 2022
• Last Update Posted: September 13, 2023
• Sponsor: BlueRock Therapeutics
• Information provided by (Responsible Party): BlueRock Therapeutics

Tracking Information

• First Submitted Date: May 3, 2022
• First Posted Date: May 5, 2022
• Last Update Posted Date: September 13, 2023
• Actual Study Start Date: July 29, 2022
• Estimated Primary Completion Date: October 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 3, 2022)

• Change in Good ON-time as measured by the PD Diary. [ Time Frame: 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.
• Individual participant variability for ON-time without troublesome dyskinesia (Good ON-time) as measured by the PD diary. [ Time Frame: 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.
• Proportion of valid PD Diaries. [ Time Frame: Baseline, 3, 6, 12, 18 and 24 months. ]
  Proportion of valid PD Diaries.

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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
• 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.
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Probability Sample
• Study Population: Parkinson's disease patients whose medications do not provide adequate control of their symptoms.
• Condition: Parkinson's Disease
• Intervention: Not Provided

Study Groups/Cohorts

• 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.

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: August 7, 2023): 400
• Original Estimated Enrollment (submitted: May 3, 2022): 500
• Estimated Study Completion Date: October 2026
• Estimated Primary Completion Date: October 2025 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 39 Years to 70 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: Not Provided

Contacts

Contact: Marta E Farino-Silva; 1-617-930-9292; clinicaltrials@bluerocktx.com

• Listed Location Countries: Canada, Germany, Italy, Spain, United States

Administrative Information

• NCT Number: NCT05363046
• Other Study ID Numbers: BRT-DA01-NIS-001
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: BlueRock Therapeutics
• Original Responsible Party: Same as current
• Current Study Sponsor: BlueRock Therapeutics
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: BlueRock Therapeutics
• Verification Date: September 2023