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

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ClinicalTrials.gov Identifier: NCT04802733
Recruitment Status : Recruiting
First Posted : March 17, 2021
Last Update Posted : September 1, 2021
Sponsor:
Collaborator:
Memorial Sloan Kettering Cancer Center
Information provided by (Responsible Party):
BlueRock Therapeutics

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.

Condition or disease Intervention/treatment Phase
Advanced Parkinson's Disease Biological: MSK-DA01 Device: MSK-DA01 Cell Delivery Device Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : May 3, 2021
Estimated Primary Completion Date : January 2023
Estimated Study Completion Date : January 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: MSK-DA01 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.




Primary Outcome Measures :
  1. 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;


Secondary Outcome Measures :
  1. 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

  2. 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.

  3. 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.

  4. 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.



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Ages Eligible for Study:   50 Years to 76 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 50-76 years old (Canada)
  • Age 60-76 years old (United States)
  • Diagnosis of Parkinson's Disease made between 5 to 15 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

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): NCT04802733


Contacts
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Contact: Jeffrey Lickteig 331-401-1444 Jeffrey.Lickteig@premier-research.com

Locations
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United States, California
University of California Irvine Recruiting
Orange, California, United States, 92868
Contact: Alexandria Murry    714-456-5956    admurry@hs.uci.edu   
United States, New York
Weill Cornell Medical College Recruiting
New York, New York, United States, 10065
Contact: Gabrielle Auerbach    212-746-2474    gra2012@med.cornell.edu   
Canada, Ontario
Toronto Western Hospital Recruiting
Toronto, Ontario, Canada, M5T 2S8
Contact: Martha Lenis, BHA, CHIM, CCRP    416-603-5800 ext 2797    Martha.Lenis@uhnresearch.ca   
Sponsors and Collaborators
BlueRock Therapeutics
Memorial Sloan Kettering Cancer Center
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Responsible Party: BlueRock Therapeutics
ClinicalTrials.gov Identifier: NCT04802733    
Other Study ID Numbers: MSK-DA01-101
First Posted: March 17, 2021    Key Record Dates
Last Update Posted: September 1, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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