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

BlueRock Therapeutics

Status and phase

Completed

Phase 1

Conditions

Advanced Parkinson's Disease

Treatments

Device: MSK-DA01 Cell Delivery Device

Biological: MSK-DA01

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT04802733

MSK-DA01-101

Details and patient eligibility

About

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.

Full 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.

Enrollment

12 patients

Sex

All

Ages

50 to 78 years old

Volunteers

No 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