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

Advanced Parkinson's Disease Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04802733
• Other study ID #: MSK-DA01-101
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: May 3, 2021
• Est. completion date: May 2024

Study information

• Verified date: May 2022
• Source: BlueRock Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 12
• Est. completion date: May 2024
• Est. primary completion date: May 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 50 Years to 78 Years

Eligibility

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

Related Conditions & MeSH terms

• Advanced Parkinson's Disease
• Parkinson 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.

Locations

Country	Name	City	State
Canada	Toronto Western Hospital	Toronto	Ontario
United States	Weill Cornell Medical College	New York	New York
United States	University of California Irvine	Orange	California

Sponsors (2)

Lead Sponsor	Collaborator
BlueRock Therapeutics	Memorial Sloan Kettering Cancer Center

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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;	Baseline to 1 Year Post-Transplant
Secondary	Evidence of Cell Survival	Change in 18F-DOPA uptake using positron emission tomography (PET) from baseline to 1 and 2 years	Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Secondary	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.	Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Secondary	Changes in Waking Hours in "Off" State	Changes in number of waking hours in the "off" state from baseline to 2 years post-transplant.	Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Secondary	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.	Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant