Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson's Disease

• ClinicalTrials.gov Identifier: NCT04802733
• Recruitment Status: Active, not recruiting
• First Posted: March 17, 2021
• Last Update Posted: June 2, 2022
• Sponsor: BlueRock Therapeutics
• Collaborator: Memorial Sloan Kettering Cancer Center
• Information provided by (Responsible Party): BlueRock Therapeutics

Tracking Information

• First Submitted Date: March 12, 2021
• First Posted Date: March 17, 2021
• Last Update Posted Date: June 2, 2022
• Actual Study Start Date: May 3, 2021
• Estimated Primary Completion Date: May 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 22, 2021)

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;

Original Primary Outcome Measures (submitted: March 15, 2021)

• incidence of SAEs Cohort A [ Time Frame: at 1-year post-transplant ]
  Safety will be defined as 2 or less of Cohort A or Cohort B developing 2 or more SAEs related to surgery, presence of transplanted cells, or immunosuppression; as 2 or less of Cohort A or Cohort B developing a tumor or abnormal tissue overgrowth related to presence of transplanted cells;
• incidence of SAEs Cohort B [ Time Frame: 1 year post-transplant ]
  Safety will be defined as 2 or less of Cohort A or Cohort B developing 2 or more SAEs related to surgery, presence of transplanted cells, or immunosuppression; as 2 or less of Cohort A or Cohort B developing a tumor or abnormal tissue overgrowth related to presence of transplanted cells;

Current Secondary Outcome Measures (submitted: March 22, 2021)

• Evidence of Cell Survival [ Time Frame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant ]
  Change in 18F-DOPA uptake using positron emission tomography (PET) from baseline to 1 and 2 years
• Changes in Motor Function [ Time Frame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant ]
  Changes in MDS-Unified Parkinson's Disease Rating Scale (UPDRS) motor sub-score in the "off" state from 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 ]
  Changes in number of waking hours in the "off" state from 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 ]
  Incidence of SAEs at 2 years post-transplant and incidence and type of AEs at 1 and 2 years post-transplant.

Original Secondary Outcome Measures (submitted: March 15, 2021)

Change in striatal 18F-DOPA uptake using positron emission tomography (PET) [ Time Frame: up to to 2 years ]

Change in striatal 18F-DOPA uptake using positron emission tomography (PET) from baseline to 1 and 2 years;

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson's Disease
• 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
• Brief 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.
• 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 Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Advanced Parkinson's Disease

Intervention

• Biological: MSK-DA01
  MSK-DA01 is an experimental product derived from human embryonic stem cells. The stem cells were converted into brain cells that produce dopamine.
• Device: MSK-DA01 Cell Delivery Device
  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 Arms

Experimental: MSK-DA01

Interventions:

• Biological: MSK-DA01
• Device: MSK-DA01 Cell Delivery Device

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: November 10, 2021): 12
• Original Estimated Enrollment (submitted: March 15, 2021): 10
• Estimated Study Completion Date: May 2024
• Estimated Primary Completion Date: May 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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
• 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 50 Years to 78 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada, United States

Administrative Information

• NCT Number: NCT04802733
• Other Study ID Numbers: MSK-DA01-101
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: Yes

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: BlueRock Therapeutics
• Original Responsible Party: Memorial Sloan Kettering Cancer Center
• Current Study Sponsor: BlueRock Therapeutics
• Original Study Sponsor: Memorial Sloan Kettering Cancer Center
• Collaborators: Memorial Sloan Kettering Cancer Center
• Investigators: Not Provided
• PRS Account: BlueRock Therapeutics
• Verification Date: May 2022