Advanced Parkinson's DiseasePhase 1NCT04802733
Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson's Disease
A Phase 1 study testing MSK-DA01 and MSK-DA01 Cell Delivery Device in people with Advanced Parkinson's Disease, run by BlueRock Therapeutics at 3 sites in 2 countries.
Trial summary
This clinical trial is designed to test whether surgically injecting nerve cells that make dopamine into the brain of Parkinson's disease patients is safe, and to monitor for potential side effects. Read more about this study
- Who is it for?
- Ages 50 to 78 years
- Conditions the study is for
- Advanced Parkinson's Disease
- What is being tested?
- MSK-DA01 and MSK-DA01 Cell Delivery Device
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 3 May 2021to 10 Jun 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
Who may be able to join
- Age 50-78 years old (Canada)
- Age 60-78 years old (United States)
- Diagnosis of Parkinson's Disease made between 3 to 20 years ago
- Taking levodopa, but with complications of therapy such as wearing off and/or dyskinesia
- Able to participate in all study visits and evaluations, including brain MRI and PET scan
- Existence of a study partner who may act as potential surrogate over long term for ongoing consent
Who cannot join
- Diagnosis of primary mitochondrial disorder, epilepsy, stroke, multiple sclerosis or other neurodegenerative diseases such as Alzheimer's disease
- Prior Deep Brain Stimulation , lesion therapy, or gene therapy for PD
- Prior surgical or radiation therapy to the brain or spinal cord
- Any medical condition resulting in high risk of immunosuppressive drugs, including any active infectious disease
- Inability to temporarily stop anti-platelet agents or other anti-coagulant medications without serious risk
- Previous or currently active malignant disease within the past 5 years, except basal cell carcinoma or in situ uterine cervical carcinoma that have been treated
Show all 10Show fewer
- Severe obesity (>350 lbs) or any condition that prevents use of PET/MRI
- Pregnancy or breastfeeding
- Contraindication to surgery or general anesthesia
- In the opinion of the investigator, any other condition regarded as making subject unsuitable for trial
Trial locations and contacts
3 sites in 2 countries.
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: Phase 1 Study To Assess the Safety and Tolerability of Human Embryonic Stem Cell-Derived Midbrain Dopamine Neuron Cell Therapy (MSK-DA01) For Advanced Parkinson's Disease
This clinical trial is designed to test whether surgically injecting nerve cells that make dopamine into the brain of Parkinson's disease patients is safe, and to monitor for potential side effects.
Read the detailed description
Subjects will undergo surgical transplantation of the dopamine-producing cells under general anesthesia into a part of the brain called the putamen. Subjects then take medicines to partially suppress their immune system (aimed to prevent the body from rejecting the cells) for 1 year. Safety, tolerability, evidence of cell survival (using MRI and PET scans of the brain), and effect on Parkinson's disease symptoms are assessed for 2 years post-transplant.
- Conditions
- Advanced Parkinson's Disease
- Being tested
- MSK-DA01, MSK-DA01 Cell Delivery Device
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MSK-DA01Biological
MSK-DA01 is an experimental product derived from human embryonic stem cells. The stem cells were converted into brain cells that produce dopamine.
MSK-DA01 Cell Delivery DeviceDevice
A device that is used for injection of fluids into the brain will be used. Some minor modifications have been made to the device to allow delivery of MSK-DA01 cells.
| Study group | Role | What they receive |
|---|---|---|
| MSK-DA01 | Experimental | Not described Receives: Biological: MSK-DA01; Device: MSK-DA01 Cell Delivery Device |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability
The incidence of Serious Adverse Events (SAEs) at 1 year post-transplant. or abnormal tissue overgrowth related to presence of transplanted cells;
Measured: Baseline to 1 Year Post-Transplant
Secondary outcomes (4)
Evidence of Cell Survival
Change in 18F-DOPA uptake using positron emission tomography (PET) from baseline to 1 and 2 years
Measured: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Changes in Motor Function
Changes in MDS-Unified Parkinson's Disease Rating Scale (UPDRS) motor sub-score in the "off" state from baseline to 2 years post-transplant.
Measured: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Changes in Waking Hours in "Off" State
Changes in number of waking hours in the "off" state from baseline to 2 years post-transplant.
Measured: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Continued Safety and Tolerability
Incidence of SAEs at 2 years post-transplant and incidence and type of AEs at 1 and 2 years post-transplant.
Measured: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2021
- Study start3 May 2021
- Primary completion18 May 2023
- Study completion10 Jun 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Parkinson's 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- BlueRock Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Memorial Sloan Kettering Cancer Center
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04802733
- Sponsor's protocol ID
- MSK-DA01-101
- Status last verified
- Oct 2024
- First submitted
- 12 Mar 2021
- Last updated
- 1 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Study To Assess the Safety and Tolerability of Human Embryonic Stem Cell-Derived Midbrain Dopamine Neuron Cell Therapy (MSK-DA01) For Advanced Parkinson's Disease
- ClinicalTrials.gov ID
- NCT04802733
- Acronym / sponsor ID
- MSK-DA01-101
- Conditions
- Advanced Parkinson's Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- BlueRock TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Memorial Sloan Kettering Cancer Center
- Study start
- 3 May 2021 (actual)
- Primary completion
- 18 May 2023 (actual)
- Study completion
- 10 Jun 2024 (actual)
- First posted
- 17 Mar 2021 (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 clinical trial is designed to test whether surgically injecting nerve cells that make dopamine into the brain of Parkinson's disease patients is safe, and to monitor for potential side effects.
Detailed description
Subjects will undergo surgical transplantation of the dopamine-producing cells under general anesthesia into a part of the brain called the putamen. Subjects then take medicines to partially suppress their immune system (aimed to prevent the body from rejecting the cells) for 1 year. Safety, tolerability, evidence of cell survival (using MRI and PET scans of the brain), and effect on Parkinson's disease symptoms are assessed for 2 years post-transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MSK-DA01 | Experimental | Biological: MSK-DA01, Device: MSK-DA01 Cell Delivery Device |
Who may qualify
- Ages
- 50 to 78 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age 50-78 years old (Canada)
- Age 60-78 years old (United States)
- Diagnosis of Parkinson's Disease made between 3 to 20 years ago
- Taking levodopa, but with complications of therapy such as wearing off and/or dyskinesia
- Able to participate in all study visits and evaluations, including brain MRI and PET scan
Key exclusion criteria (10 listed)
- Diagnosis of primary mitochondrial disorder, epilepsy, stroke, multiple sclerosis or other neurodegenerative diseases such as Alzheimer's disease
- Prior Deep Brain Stimulation , lesion therapy, or gene therapy for PD
- Prior surgical or radiation therapy to the brain or spinal cord
- Any medical condition resulting in high risk of immunosuppressive drugs, including any active infectious disease
- Inability to temporarily stop anti-platelet agents or other anti-coagulant medications without serious risk
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MSK-DA01MSK-DA01 is an experimental product derived from human embryonic stem cells. The stem cells were converted into brain cells that produce dopamine. | Biological | MSK-DA01 |
| MSK-DA01 Cell Delivery DeviceA device that is used for injection of fluids into the brain will be used. Some minor modifications have been made to the device to allow delivery of MSK-DA01 cells. | Device | MSK-DA01 |
Primary outcome
Safety and Tolerability
Time frame: Baseline to 1 Year Post-Transplant
The incidence of Serious Adverse Events (SAEs) at 1 year post-transplant. or abnormal tissue overgrowth related to presence of transplanted cells;
4 secondary outcomes
Evidence of Cell Survival
Time frame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Changes in Motor Function
Time frame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Changes in Waking Hours in "Off" State
Time frame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Continued Safety and Tolerability
Time frame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Nov 2024.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MSK-DA01 and MSK-DA01 Cell Delivery Device in people with Advanced Parkinson's 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 6 inclusion and 10 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: finished, as of the sponsor's last update on 1 Nov 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety and Tolerability (time frame: Baseline to 1 Year Post-Transplant). 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 1 Nov 2024.