Continued Evaluation of Patients With Parkinson's Disease Who Previously Received BRT-DA01

NCT05897957 | Enrolling By Invitation

• 1 other identifier: BRT-DA01-501
• Study Type: observational
• Target: 12
• Locations: 2 countries
• Sites: 3

Brief Summary

This is a multicenter study to evaluate the safety and clinical outcomes of BRT-DA01 in subjects with PD who previously received BRT-DA01 in the Phase 1 Study MSK-DA01-101.No investigational therapy will be administered in this study.

Conditions

Parkinson Disease

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Total SAEs and total number of subjects with at least 1 SAE

  Total SAEs and total number of subjects with at least 1 SAE related to cellular drug product

  Baseline through 5 years post-transplantation

Secondary Outcomes (6)

• Change in striatal 18F-DOPA uptake using positron emission tomography (PET)

  Baseline through 5 years post-transplantation

• Change in Hauser PD diary measurements

  Baseline through 5 years post-transplantation

• Change in the International Parkinson and Movement Disorder Society (MDS)-Unified Parkinson's Disease Rating Scale (UPDRS)

  Baseline through 5 years post-transplantation

• Change in levodopa equivalent daily dose (LEDD)

  Baseline through 5 years post-transplantation

• Change in Parkinson's Disease Questionnaire-39 (PDQ-39) summary index

  Baseline through 5 years post-transplantation

Other Outcomes (4)

• Adverse Events

  Baseline through 5 years post-transplantation

• Change in modified Schwab and England Activities of Daily Living (mSEADL) scale

  Baseline through 5 years post-transplantation

• Change in brain atrophy using MRI

  Baseline through 5 years post-transplantation

Eligibility Criteria

• Age: 50 Years - 78 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Advanced Parkinson's Disease who previously received BRT-DA01 in the Phase 1 Study MSK-DA01-101

You may qualify if:

• Subject received BRT-DA01 in Study MSK-DA01-101 (ie, parent study) and will have completed or discontinued the parent study
• Subject must be willing and able to provide written informed consent for the BRT-DA0-501 study in accordance with applicable regulations and guidelines and to comply with all study visits and procedures

Sponsors & Collaborators

• BlueRock Therapeutics: lead

Study Sites (3)

University of California, Irvine

Orange, California, 92868, United States

Location

Weill Cornell Medical College

New York, New York, 10065, United States

Location

Toronto Western Hospital

Toronto, Ontario, M5T 2S8, Canada

Location

Biospecimen

Retention: SAMPLES WITH DNA

Biomarkers for PD may be explored

MeSH Terms

Conditions

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

Condition Hierarchy (Ancestors)

Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Nauman Abid, MD

  BlueRock Therapeutics LP

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: June 1, 2023
• First Posted: June 12, 2023
• Study Start: May 16, 2023
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): September 1, 2027
• Last Updated: April 12, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2); CA (1)