Clinical trial NCT05897957: Continued Evaluation of Patients With Parkinson's Disease Who Previously Received BRT-DA01
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
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
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
Lead sponsor: BlueRock Therapeutics
Sponsor class: INDUSTRY
First posted: 2023-06-12
Enrollment: 12
Sex: ALL
Minimum age (years): 50
Maximum age (years): 78
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Advanced Parkinson's Disease who previously received BRT-DA01 in the Phase 1 Study MSK-DA01-101

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.

Eligibility criteria: 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

Primary outcomes:
measure: Total SAEs and total number of subjects with at least 1 SAE
timeFrame: Baseline through 5 years post-transplantation
Secondary outcomes:
measure: Change in striatal 18F-DOPA uptake using positron emission tomography (PET)
timeFrame: Baseline through 5 years post-transplantation
measure: Change in Hauser PD diary measurements
timeFrame: Baseline through 5 years post-transplantation
measure: Change in the International Parkinson and Movement Disorder Society (MDS)-Unified Parkinson's Disease Rating Scale (UPDRS)
timeFrame: Baseline through 5 years post-transplantation
measure: Change in levodopa equivalent daily dose (LEDD)
timeFrame: Baseline through 5 years post-transplantation
measure: Change in Parkinson's Disease Questionnaire-39 (PDQ-39) summary index
timeFrame: Baseline through 5 years post-transplantation
measure: Change in the Unified Dyskinesia Rating Scale (UDysRS)
timeFrame: Baseline through 5 years post-transplantation
Other outcomes:
measure: Adverse Events
timeFrame: Baseline through 5 years post-transplantation
measure: Change in modified Schwab and England Activities of Daily Living (mSEADL) scale
timeFrame: Baseline through 5 years post-transplantation
measure: Change in brain atrophy using MRI
timeFrame: Baseline through 5 years post-transplantation
measure: Subjects using alternative advanced treatment options in Parkin's Disease
timeFrame: Baseline through 5 years post-transplantation
Locations:
city: Orange
country: United States
facility: University of California, Irvine
state: California
city: New York
country: United States
facility: Weill Cornell Medical College
state: New York
city: Toronto
country: Canada
facility: Toronto Western Hospital
state: Ontario
Study officials:
affiliation: BlueRock Therapeutics LP
name: Nauman Abid, MD
role: STUDY_DIRECTOR
References: