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

Primary Purpose

Advanced Parkinson's Disease

Status
Active
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
MSK-DA01
MSK-DA01 Cell Delivery Device
Sponsored by
BlueRock Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Advanced Parkinson's Disease

Eligibility Criteria

50 Years - 78 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of California Irvine
  • Weill Cornell Medical College
  • Toronto Western Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

MSK-DA01

Arm Description

Outcomes

Primary Outcome Measures

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;

Secondary Outcome Measures

Evidence of Cell Survival
Change in 18F-DOPA uptake using positron emission tomography (PET) from baseline to 1 and 2 years
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.
Changes in Waking Hours in "Off" State
Changes in number of waking hours in the "off" state from 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.

Full Information

First Posted
March 12, 2021
Last Updated
May 31, 2022
Sponsor
BlueRock Therapeutics
Collaborators
Memorial Sloan Kettering Cancer Center
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1. Study Identification

Unique Protocol Identification Number
NCT04802733
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
May 3, 2021 (Actual)
Primary Completion Date
May 2023 (Anticipated)
Study Completion Date
May 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
BlueRock Therapeutics
Collaborators
Memorial Sloan Kettering Cancer Center

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
Yes
Data Monitoring Committee
Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Advanced Parkinson's Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
MSK-DA01
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
MSK-DA01
Intervention Description
MSK-DA01 is an experimental product derived from human embryonic stem cells. The stem cells were converted into brain cells that produce dopamine.
Intervention Type
Device
Intervention Name(s)
MSK-DA01 Cell Delivery Device
Intervention Description
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 Measure Information:
Title
Safety and Tolerability
Description
The incidence of Serious Adverse Events (SAEs) at 1 year post-transplant. or abnormal tissue overgrowth related to presence of transplanted cells;
Time Frame
Baseline to 1 Year Post-Transplant
Secondary Outcome Measure Information:
Title
Evidence of Cell Survival
Description
Change in 18F-DOPA uptake using positron emission tomography (PET) from baseline to 1 and 2 years
Time Frame
Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Title
Changes in Motor Function
Description
Changes in MDS-Unified Parkinson's Disease Rating Scale (UPDRS) motor sub-score in the "off" state from baseline to 2 years post-transplant.
Time Frame
Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Title
Changes in Waking Hours in "Off" State
Description
Changes in number of waking hours in the "off" state from baseline to 2 years post-transplant.
Time Frame
Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Title
Continued Safety and Tolerability
Description
Incidence of SAEs at 2 years post-transplant and incidence and type of AEs at 1 and 2 years post-transplant.
Time Frame
Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant

10. Eligibility

Sex
All
Minimum Age & Unit of Time
50 Years
Maximum Age & Unit of Time
78 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
University of California Irvine
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Weill Cornell Medical College
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Toronto Western Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5T 2S8
Country
Canada

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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