History of Changes for Study: NCT04802733

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

Latest version (submitted May 31, 2022) on ClinicalTrials.gov

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Study Record Versions

Version	Submitted Date	Changes
1	March 15, 2021	None (earliest Version on record)
2	March 22, 2021	Contacts/Locations, Outcome Measures, Arms and Interventions, Study Status, Study Identification, Sponsor/Collaborators, IPDSharing, Conditions, Study Description, Oversight, References, Eligibility and Study Design
3	March 26, 2021	Recruitment Status, Study Status and Contacts/Locations
4	May 10, 2021	Study Status
5	June 9, 2021	Contacts/Locations, Study Status and Eligibility
6	June 21, 2021	Contacts/Locations and Study Status
7	August 23, 2021	Contacts/Locations and Study Status
8	August 31, 2021	Contacts/Locations and Study Status
9	October 25, 2021	Contacts/Locations and Study Status
10	November 10, 2021	Study Status and Study Design
11	January 6, 2022	Contacts/Locations and Study Status
12	March 23, 2022	Study Status and Eligibility
13	May 31, 2022	Recruitment Status, Study Status, Contacts/Locations and Study Design

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Study NCT04802733
Submitted Date: June 21, 2021 (v6)

Study Identification


Unique Protocol ID:	MSK-DA01-101
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
Secondary IDs:

Study Status


Record Verification:	June 2021
Overall Status:	Recruiting
Study Start:	May 3, 2021
Primary Completion:	January 2023 [Anticipated]
Study Completion:	January 2024 [Anticipated]

First Submitted:	March 12, 2021
First Submitted that Met QC Criteria:	March 15, 2021
First Posted:	March 17, 2021 [Actual]

Last Update Submitted that Met QC Criteria:	June 21, 2021
Last Update Posted:	June 23, 2021 [Actual]

Sponsor/Collaborators


Sponsor:	BlueRock Therapeutics
Responsible Party:	Sponsor
Collaborators:	Memorial Sloan Kettering Cancer Center

Oversight


U.S. FDA-regulated Drug:	Yes
U.S. FDA-regulated Device:	Yes
Unapproved/Uncleared Device:
Pediatric Postmarket Surveillance:
Data Monitoring:	Yes

Study Description

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 Design


Study Type:	Interventional
Primary Purpose:	Treatment
Study Phase:	Phase 1
Interventional Study Model:	Single Group Assignment
Number of Arms:	1
Masking:	None (Open Label)
Allocation:	N/A
Enrollment:	10 [Anticipated]

Arms and Interventions

Arms	Assigned Interventions
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.

Outcome Measures


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.

Eligibility


Minimum Age:	50 Years
Maximum Age:	76 Years
Sex:	All
Gender Based:
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

Contacts/Locations


Central Contact Person:	Jeffrey Lickteig Telephone: 331-401-1444 Email: Jeffrey.Lickteig@premier-research.com
Locations:	United States, California
	University of California Irvine [Recruiting] Orange, California, United States, 92868 Contact: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:Contact: Gabrielle Auerbach 212-746-2474 gra2012@med.cornell.edu
	Canada, Ontario
	Toronto Western Hospital [Not yet recruiting] Toronto, Ontario, Canada, M5T 2S8 Contact:Contact: Martha Lenis, BHA, CHIM, CCRP 516-858-6878 Martha.Lenis@uhnresearch.ca

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