Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson's Disease
|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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04802733|
Recruitment Status : Recruiting
First Posted : March 17, 2021
Last Update Posted : January 10, 2022
|Condition or disease||Intervention/treatment||Phase|
|Advanced Parkinson's Disease||Biological: MSK-DA01 Device: MSK-DA01 Cell Delivery Device||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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 :||March 2023|
|Estimated Study Completion Date :||March 2024|
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.
- 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;
- 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.
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
|Contact: Jeffrey Lickteig||331-401-1444||Jeffrey.Lickteig@premier-research.com|
|United States, California|
|University of California Irvine||Recruiting|
|Orange, California, United States, 92868|
|Contact: Jennifer Scott 714-456-5956 Scottj2@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 firstname.lastname@example.org|
|Toronto Western Hospital||Recruiting|
|Toronto, Ontario, Canada, M5T 2S8|
|Contact: Martha Lenis, BHA, CHIM, CCRP 416-603-5800 ext 2797 Martha.Lenis@uhnresearch.ca|