Parkinson DiseaseNCT05897957
Continued Evaluation of Patients With Parkinson's Disease Who Previously Received BRT-DA01
An observational study of Parkinson Disease, run by BlueRock Therapeutics at 3 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 50 to 78 years
- Conditions the study is for
- Parkinson Disease
- How many people
- 12 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 16 May 2023to Sep 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 to 78 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Advanced Parkinson's Disease who previously received BRT-DA01 in the Phase 1 Study MSK-DA01-101
Who may be able to join
- 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
Who cannot join
- No exclusion criteria
Trial locations and contacts
3 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
3 sites in 2 countries
United States2 sites
Weill Cornell Medical College
New York, New York, 10065
University of California, Irvine
Orange, California, 92868
About this study
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
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.
Read the 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.
- 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
- Medical subject headings
- Parkinson Disease, Parkinsonian Disorders, Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Movement Disorders, Synucleinopathies
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline through 5 years post-transplantation
Secondary outcomes (6)
Change in striatal 18F-DOPA uptake using positron emission tomography (PET)
Change in striatal 18F-DOPA uptake using positron emission tomography (PET)
Measured: Baseline through 5 years post-transplantation
Change in Hauser PD diary measurements
Change in Hauser PD diary measurements
Measured: 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
Measured: Baseline through 5 years post-transplantation
Change in levodopa equivalent daily dose (LEDD)
Change in LEDD
Measured: Baseline through 5 years post-transplantation
Change in Parkinson's Disease Questionnaire-39 (PDQ-39) summary index
Change in PDQ-39
Measured: Baseline through 5 years post-transplantation
Change in the Unified Dyskinesia Rating Scale (UDysRS)
Change in UDysRS
Measured: Baseline through 5 years post-transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2023
- Study start16 May 2023
- Primary completionMay 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BlueRock Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Nauman Abid, MD, Study director, BlueRock Therapeutics LP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05897957
- Sponsor's protocol ID
- BRT-DA01-501
- Status last verified
- Apr 2024
- First submitted
- 1 Jun 2023
- Last updated
- 12 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05897957
- Acronym / sponsor ID
- BRT-DA01-501
- Conditions
- Parkinson Disease
- Keywords
- Parkinson Disease, Parkinsonian Disorders, Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Movement Disorders, Synucleinopathies
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- BlueRock TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 16 May 2023 (actual)
- Primary completion
- May 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 12 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 50 to 78 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Advanced Parkinson's Disease who previously received BRT-DA01 in the Phase 1 Study MSK-DA01-101
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (1 listed)
- No exclusion criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
6 secondary outcomes
Change in striatal 18F-DOPA uptake using positron emission tomography (PET)
Time frame: Baseline through 5 years post-transplantation
Change in Hauser PD diary measurements
Time frame: Baseline through 5 years post-transplantation
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 levodopa equivalent daily dose (LEDD)
Time frame: Baseline through 5 years post-transplantation
Change in Parkinson's Disease Questionnaire-39 (PDQ-39) summary index
Time frame: Baseline through 5 years post-transplantation
Change in the Unified Dyskinesia Rating Scale (UDysRS)
Time frame: Baseline through 5 years post-transplantation
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Apr 2024.
- Individual participant dataSharing planned: No
Related trials for Parkinson Disease
Not yet recruitingPhase 1/2
STAR0602 With Nivolumab in Non Small Cell Lung Cancer and in Metastatic Castrate-Resistant Prostate Cancer, and STAR0602 Alone in Thymic Carcinoma
NSCLC, Non-Small Cell Lung Cancer
Enrolling by invitationObservational study
Early Molecular Biomarkers for Differentiating Parkinsonian Syndromes
PARKINSON DISEASE (Disorder), Atypical Parkinsonism
RecruitingPhase 3
A Clinical Study of Sacituzumab Tirumotecan (MK-2870) in Combination With Pembrolizumab (MK-3475) as First-line Maintenance Treatment of Cervical Cancer (MK-2870-036/TroFuse-036/GOG-3123/ENGOT-cx22)
Cervical Cancer
Active, not recruitingPhase 1/2
Clinical Safety and Efficacy of Umbilical Cord Mesenchymal Stromal Cells-derived Exosomes in Patients With Parkinson's Disease
Parkinson Disease (PD)
Active, not recruitingObservational study
Adaptive DBS to Offset STN Dysregulation During Exercise in PD
Parkinson Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Nauman Abid, MD study director
BlueRock Therapeutics LP
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Parkinson Disease, run by BlueRock Therapeutics at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 12 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total SAEs and total number of subjects with at least 1 SAE (time frame: Baseline through 5 years post-transplantation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Apr 2024.