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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
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.
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
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.
Time frame: 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.
Time frame: From baseline to 2 years post surgery
18F-FP-CIT uptake using positron emission tomography (PET)
Changes in 18F-FP-CIT uptake using positron emission tomography (PET) from baseline.
Time frame: From baseline to 2 years post surgery
The situation of implantation and overgrowth of transplanted cells using cranial MRI
Volume changes of transplanted cells using cranial magnetic resonance imaging (MRI)
Time frame: 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.
Time frame: 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.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Minimum score: 0; Maximum score: 52; Higher scores mean a worse outcome.
Time frame: 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
Time frame: From baseline to 2 years post surgery
Changes in daily levodopa equivalent dose (LED) from baseline.
Time frame: 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.
Time frame: From baseline to 2 years post surgery