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

Sponsor

BlueRock Therapeutics (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT05897957

Collaborator

(none)

Enrollment

Location

51.5

Anticipated Duration (Months)

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Parkinson Disease

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 Design

Study Type:

Observational

Anticipated Enrollment :

12 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Continued Evaluation of Patients With Parkinson's Disease Who Previously Received BRT-DA01, a Human Embryonic Stem Cell-Derived Midbrain Dopaminergic Neuronal Cell Therapy

Actual Study Start Date :

May 16, 2023

Anticipated Primary Completion Date :

May 1, 2027

Anticipated Study Completion Date :

Sep 1, 2027

Outcome Measures

Primary Outcome Measures

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

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 Hauser PD diary measurements [Baseline through 5 years post-transplantation]
Change in Hauser PD diary measurements
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
Change in the Unified Dyskinesia Rating Scale (UDysRS) [Baseline through 5 years post-transplantation]
Change in UDysRS

Other Outcome Measures

Adverse Events [Baseline through 5 years post-transplantation]
AEs related to the cellular drug product
Change in modified Schwab and England Activities of Daily Living (mSEADL) scale [Baseline through 5 years post-transplantation]
Change in mSEADL
Change in brain atrophy using MRI [Baseline through 5 years post-transplantation]
Change in brain atrophy using MRI
Subjects using alternative advanced treatment options in Parkin's Disease [Baseline through 5 years post-transplantation]
Number of subjects using alternative advanced treatment options in PD (eg, CSAI, LCIG, DBS, focused ultrasound ablation)

Eligibility Criteria

Criteria

Ages Eligible for Study:

50 Years to 78 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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