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| Name | Class |
|---|---|
| California Institute for Regenerative Medicine (CIRM) | OTHER |
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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)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ANPD001 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ANPD001 | Biological | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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) | 1 year (primary follow up) |
| Measure | Description | Time Frame |
|---|---|---|
| "ON" time without troublesome dyskinesia | 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) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Revati Shreeniwas, MD, FCCP | Aspen Neuroscience | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Arizona - Banner Health | Tucson | Arizona | 85724 | United States | ||
| Scripps Health |
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| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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Open label, dose escalation of bilateral injection of ANPD001 dopaminergic precursor cells to the putamen
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| Custom Device | Device | A custom device to facilitate slow injection of small volumes of investigational drugs (including cell therapies), under MRI guidance |
|
Part II score range is 0 to 52 with 0 being normal and 30 and above being severe |
| 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 | 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) | 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) | 14 years of long term follow up (4 years in-person visits and 10 additional years follow up via telephone call) |
| La Jolla |
| California |
| 92037 |
| United States |
| University of California, Los Angeles | Los Angeles | California | 90095 | United States |
| University of California, Irvine | Orange | California | 92868 | United States |
| University of California, San Diego | San Diego | California | 92037 | United States |
| University of California, San Francisco | San Francisco | California | 94143 | United States |
| University of Colorado | Aurora | Colorado | 80045 | United States |
| Emory University | Atlanta | Georgia | 30322 | United States |
| Feinstein Institutes | Manhasset | New York | 11030 | United States |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |