This clinical trial is designed to evaluate the safety, tolerability and preliminary efficacy of a single injection of NouvNeu001 (Human Dopaminergic Progenitor Cells Injection) in patients with Multiple System Atrophy.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
9
Single injection of Human Dopaminergic Progenitor Cells into the putamen/striatum region of brain.
Beijing Tiantan Hospital, Capital Medical University
Beijing, Beijing Municipality, China
Incidence and severity of Adverse Events as Assessed by CTCAE V5.0
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs).
Time frame: 52 weeks post-transplant
Change from baseline in the UMSARS total score (Part I + Part II) at Weeks 13, 26, and 52.
Change from baseline in the Unified Multiple System Atrophy Rating Scale (UMSARS) total score (Part I Score 0 \~ 48 + Part II Score 0 \~ 56; ) at Weeks 13, 26, and 52. A higher score indicates more severe symptoms or worse health.
Time frame: Weeks 13, 26, and 52 post-transplant
Change from baseline in the UMSARS Part I score at Weeks 13, 26, and 52.
Change from baseline in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part I score (0 \~ 48) at Weeks 13, 26, and 52. A higher score indicates more severe symptoms or worse health.
Time frame: Weeks 13, 26, and 52 post-transplant
Change from baseline in the UMSARS Part II score at Weeks 13, 26, and 52.
Change from baseline in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part II score (0 \~ 56) at Weeks 13, 26, and 52. A higher score indicates more severe symptoms or worse health.
Time frame: Weeks 13, 26, and 52 post-transplant
Change from baseline in dopaminergic metabolism of the basal ganglia at Week 52, as measured by DAT PET imaging.
Change from baseline in dopaminergic metabolism of the basal ganglia at Week 52, as measured by 18F-DOPA PET or 11C-CFT DAT PET imaging.
Time frame: Weeks 52 post-transplant
Change from baseline in the CGI-S score at Weeks 13, 26, and 52.
Change from baseline in the Clinical Global Impression-Severity scale (CGI-S) score (1 \~ 7) at Weeks 13, 26, and 52. A higher score indicates more severe symptoms or worse health.
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Time frame: Weeks 13, 26, and 52 post-transplant