Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Ruijin Hospital | OTHER |
Not provided
Not provided
Not provided
Not provided
This clinical study is designed to explore the safety and tolerability of UX-DA001. It will also explore if UX-DA001 works to improve motor function in subjects with Parkinson's disease. UX-DA001 manufactured from participant's own cells will differentiate into mature dopaminergic neurons after being transplanted into the brain of the participant.
This study is an open-label, multi-center, dose-escalation and dose-expansion exploratory clinical study to evaluate the safety, tolerability, and potential efficacy of UX-DA001 Injection at different dose levels implanted in subjects with idiopathic PD.
Each subject receives only one dose of UX-DA001 for implantation into the putamen bilaterally using stereotactic neurosurgery under general anesthesia. Safety and tolerability of UX-DA001 and its effect on Parkinson's disease symptoms are assessed for 2 years post-treatment.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| UX-DA001 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| UX-DA001 | Biological | UX-DA001 (Human Midbrain Dopaminergic Progenitor Cells) is used for treating patients with iPD via implanting into bilateral putamina under stereotactic neurosurgery. Two dose levels will be planned. Each patient only receives one corresponding dose of UX-DA001. |
| Measure | Description | Time Frame |
|---|---|---|
| The incidence and and severity of adverse events (AEs) associated with surgery and/or investigational product | The incidence and and severity of adverse events (AEs) associated with surgery and/or investigational product during the surgical treatment period and the 4-week postoperative observation period. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0. | within 4 weeks post surgery |
| The incidence and severity of AEs/serious adverse events (SAEs) | The incidence and severity of AEs/serious adverse events (SAEs) during the study, including AEs and SAEs during the surgery treatment period, postoperative observation period, and follow-up period. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0. | From baseline to 2 years post surgery |
| Measure | Description | Time Frame |
|---|---|---|
| 18F-FP-CIT uptake using positron emission tomography (PET) | Changes in 18F-FP-CIT uptake using positron emission tomography (PET) from baseline. | From baseline to 2 years post surgery |
| The situation of implantation and overgrowth of transplanted cells using cranial MRI |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Jun Liu, MD, PhD | Ruijin Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ruijin Hospital, Shanghai Jiao Tong University School of Medicine | Shanghai | Shanghai Municipality | 200025 | China |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
Not provided
Not provided
open-label, multi-center, dose-escalation and dose-expansion
Not provided
Not provided
Not provided
Not provided
|
Volume changes of transplanted cells using cranial magnetic resonance imaging (MRI) |
| From baseline to 2 years post surgery |
| Changes in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score, part III, from baseline. | Minimum score: 0; Maximum score: 132; Higher scores mean a worse outcome. | From baseline to 2 years post surgery |
| Changes in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score, part II, from baseline. | Minimum score: 0; Maximum score: 52; Higher scores mean a worse outcome. | From baseline to 2 years post surgery |
| Changes in modified modified Hoehn-Yahr (H&Y) scale from baseline | Grade 0~Grade 5, lower grade means a better outcome | From baseline to 2 years post surgery |
| Changes in daily levodopa equivalent dose (LED) from baseline. | From baseline to 2 years post surgery |
| Changes in the scores of non-motor symptom scales (NMSS) from baseline. | Minimum score: 0; Maximum score: 960; Higher scores mean a worse outcome. | From baseline to 2 years post surgery |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |