This clinical trial is designed to test the safety and tolerability of injecting ANPD001 cells that will mature into dopamine-producing cells into the brain of participants with Parkinson Disease. All participants will have ANPD001 cells manufactured from their own previously collected cells.
Participants will undergo a surgical implantation of cells that will mature into dopamine-producing neurons under general anesthesia into a part of the brain where dopamine production is decreased in patients with Parkinson Disease. The effect on Parkinson Disease symptoms, safety and tolerability, and cell survival are assessed for 5 years post-transplant (with MRI and PET imaging scans of the brain). Safety and tolerability are assessed annually for an additional 10 years via telephone call (total follow-up of 15 years)
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
Biologic: ANPD001 is an experimental product derived from the patient's own skin cells converted to induced pluripotent stem cells. The stem cells were differentiated into precursors for brain cells that produce dopamine.
A custom device to facilitate slow injection of small volumes of investigational drugs (including cell therapies), under MRI guidance
University of Arizona - Banner Health
Tucson, Arizona, United States
Scripps Health
La Jolla, California, United States
University of California, Los Angeles
Los Angeles, California, United States
University of California, Irvine
Incidence and severity of treatment emergent adverse events (Safety and Tolerability)
incidence and severity of treatment emergent adverse events (TEAE) and serious adverse events (SAE)
Time frame: 1 year (primary follow up)
"ON" time without troublesome dyskinesia
Time frame: 1 year (primary follow up) and 5 years (long term follow up)
Post-injection change in Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II (Activities of Daily Living - ADL) and Part III (motor score) in the ON state (total score and scores for Parts I-IV)
Part II score range is 0 to 52 with 0 being normal and 30 and above being severe
Time frame: 1 year (primary follow up) and 5 years (long term follow up)
Post-injection change in the Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score in the practically defined OFF state
Part III score range is 0 to 132 with 0 being normal and 59 and above being severe
Time frame: 1 year (primary follow up) and 5 years (long term follow up)
Post-injection change in the 18F-DOPA uptake in the putamen
Post-injection change in the 18-fluorodopa uptake in the putamen from baseline via positron emission tomography (PET)
Time frame: 1 year (primary follow up) and 5 years (long term follow up)
Incidence and severity of treatment emergent adverse events during long term follow-up (Continued Safety and Tolerability)
Time frame: 14 years of long term follow up (4 years in-person visits and 10 additional years follow up via telephone call)
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Orange, California, United States
University of California, San Diego
San Diego, California, United States
University of California, San Francisco
San Francisco, California, United States
University of Colorado
Aurora, Colorado, United States
Emory University
Atlanta, Georgia, United States
Feinstein Institutes
Manhasset, New York, United States