Clinical trial NCT04802733: 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
Overall status: COMPLETED
Phase: PHASE1
Conditions: Advanced Parkinson's Disease
Lead sponsor: BlueRock Therapeutics
Sponsor class: INDUSTRY
Collaborators: name: Memorial Sloan Kettering Cancer Center
First posted: 2021-03-17
Enrollment: 12
Sex: ALL
Minimum age (years): 50
Maximum age (years): 78
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Safety and Tolerability
timeFrame: Baseline to 1 Year Post-Transplant
Secondary outcomes:
measure: Evidence of Cell Survival
timeFrame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
measure: Changes in Motor Function
timeFrame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
measure: Changes in Waking Hours in "Off" State
timeFrame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
measure: Continued Safety and Tolerability
timeFrame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Other outcomes:
Locations:
city: Orange
country: United States
facility: University of California Irvine
state: California
city: New York
country: United States
facility: Weill Cornell Medical College
state: New York
city: Toronto
country: Canada
facility: Toronto Western Hospital
state: Ontario
Study officials:
References: