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

Study Overview
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Brief Summary
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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
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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.

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

Conditions
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Parkinson Disease

Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 50 Years
Maximum Age: 78 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Brain Diseases, Parkinson Disease, Parkinsonian Disorders, Basal Ganglia Diseases, Central Nervous System Diseases, Nervous System Diseases, Movement Disorders, Synucleinopathies, Neurodegenerative Diseases