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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05897957
Recruitment Status : Enrolling by invitation
First Posted : June 12, 2023
Last Update Posted : April 12, 2024
Sponsor:
Information provided by (Responsible Party):
BlueRock Therapeutics

Study Description
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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
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
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Study Type : Observational
Estimated 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
Estimated Primary Completion Date : May 2027
Estimated Study Completion Date : September 2027

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. Total SAEs and total number of subjects with at least 1 SAE [ Time Frame: 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 :
  1. Change in striatal 18F-DOPA uptake using positron emission tomography (PET) [ Time Frame: Baseline through 5 years post-transplantation ]
    Change in striatal 18F-DOPA uptake using positron emission tomography (PET)

  2. Change in Hauser PD diary measurements [ Time Frame: Baseline through 5 years post-transplantation ]
    Change in Hauser PD diary measurements

  3. Change in the International Parkinson and Movement Disorder Society (MDS)-Unified Parkinson's Disease Rating Scale (UPDRS) [ Time Frame: Baseline through 5 years post-transplantation ]
    Change in the MDS-UPDRS

  4. Change in levodopa equivalent daily dose (LEDD) [ Time Frame: Baseline through 5 years post-transplantation ]
    Change in LEDD

  5. Change in Parkinson's Disease Questionnaire-39 (PDQ-39) summary index [ Time Frame: Baseline through 5 years post-transplantation ]
    Change in PDQ-39

  6. Change in the Unified Dyskinesia Rating Scale (UDysRS) [ Time Frame: Baseline through 5 years post-transplantation ]
    Change in UDysRS


Other Outcome Measures:
  1. Adverse Events [ Time Frame: Baseline through 5 years post-transplantation ]
    AEs related to the cellular drug product

  2. Change in modified Schwab and England Activities of Daily Living (mSEADL) scale [ Time Frame: Baseline through 5 years post-transplantation ]
    Change in mSEADL

  3. Change in brain atrophy using MRI [ Time Frame: Baseline through 5 years post-transplantation ]
    Change in brain atrophy using MRI

  4. Subjects using alternative advanced treatment options in Parkin's Disease [ Time Frame: Baseline through 5 years post-transplantation ]
    Number of subjects using alternative advanced treatment options in PD (eg, CSAI, LCIG, DBS, focused ultrasound ablation)


Biospecimen Retention:   Samples With DNA
Biomarkers for PD may be explored

Eligibility Criteria
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Ages Eligible for Study:   50 Years to 78 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Advanced Parkinson's Disease who previously received BRT-DA01 in the Phase 1 Study MSK-DA01-101
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05897957


Locations
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United States, California
University of California, Irvine
Orange, California, United States, 92868
United States, New York
Weill Cornell Medical College
New York, New York, United States, 10065
Canada, Ontario
Toronto Western Hospital
Toronto, Ontario, Canada, M5T 2S8
Sponsors and Collaborators
BlueRock Therapeutics
Investigators
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Study Director: Nauman Abid, MD BlueRock Therapeutics LP
More Information
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Responsible Party: BlueRock Therapeutics
ClinicalTrials.gov Identifier: NCT05897957    
Other Study ID Numbers: BRT-DA01-501
First Posted: June 12, 2023    Key Record Dates
Last Update Posted: April 12, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by BlueRock Therapeutics:
Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
 Nervous System Diseases
Movement Disorders
Synucleinopathies
Neurodegenerative Diseases
Additional relevant MeSH terms:
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Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
 Nervous System Diseases
Movement Disorders
Synucleinopathies
Neurodegenerative Diseases