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

• Conditions: Parkinson Disease

• Registration Number: NCT05897957
• Lead Sponsor: BlueRock Therapeutics

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.

Detailed Description

All subjects who received BRT-DA01 transplantation in Study MSK-DA01-101 (ie, parent study) will be asked to enroll in Study BRT-DA01-501. Subjects enrolled in this study are to be monitored for safety and clinical outcomes through 5 years post-BRT-DA01 transplantation. For individual participating subjects, the Baseline visit for the BRT-DA01-501 study will be defined as the last observation recorded in the parent study.

Study Timeline

• Study Start: 2023-05-16
• Study First Submitted: 2023-06-01
• Study First Submitted QC: 2023-06-01
• Study First Posted: 2023-06-12
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-11
• Last Update Posted: 2024-04-12
• Primary Completion: 2027-05
• Study Completion: 2027-09

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

• 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

Exclusion Criteria

• No exclusion criteria

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Total SAEs and total number of subjects with at least 1 SAE	Baseline through 5 years post-transplantation	Total SAEs and total number of subjects with at least 1 SAE related to cellular drug product

Secondary Outcome Measures

Name	Time	Method
Change in the Unified Dyskinesia Rating Scale (UDysRS)	Baseline through 5 years post-transplantation	Change in UDysRS
Change in Hauser PD diary measurements	Baseline through 5 years post-transplantation	Change in Hauser PD diary measurements
Change in striatal 18F-DOPA uptake using positron emission tomography (PET)	Baseline through 5 years post-transplantation	Change in striatal 18F-DOPA uptake using positron emission tomography (PET)
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 the MDS-UPDRS
Change in levodopa equivalent daily dose (LEDD)	Baseline through 5 years post-transplantation	Change in LEDD
Change in Parkinson's Disease Questionnaire-39 (PDQ-39) summary index	Baseline through 5 years post-transplantation	Change in PDQ-39

Trial Locations

Locations (3)

University of California, Irvine; 🇺🇸 Orange, California, United States

Toronto Western Hospital; 🇨🇦 Toronto, Ontario, Canada

Weill Cornell Medical College; 🇺🇸 New York, New York, United States