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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NouvNeu001 | Experimental | During the enrollment phase for the low-dose cohort, 3 participants will be randomized to the experimental arm to receive a single administration of NouvNeu001 Injection. |
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| control | No Intervention | During the enrollment phase for the low-dose cohort, 3 participants will be randomized to the control arm, which will not receive NouvNeu001 Injection. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Human Dopaminergic Progenitor Cells | Biological | Single injection of Human Dopaminergic Progenitor Cells into the putamen/striatum region of brain. |
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| Measure | Description | Time Frame |
|---|---|---|
| Incidence and severity of Adverse Events as Assessed by CTCAE V5.0 | Incidence and severity of adverse events (AEs) and serious adverse events (SAEs). | 52 weeks post-transplant |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Weeks 13, 26, and 52 post-transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacodynamic Biomarkers (NfL) | measurement of neurofilament light chain (NfL) levels in human blood samples. | Weeks 13, 26, and 52 post-transplant |
| Pharmacodynamic Biomarkers (α-syn) | measurement of alpha-synuclein (α-syn) levels in human blood samples. |
Inclusion Criteria:
Exclusion Criteria:
Speech impairment defined by a score of ≥3 on UMSARS Item 1. Swallowing impairment defined by a score of ≥3 on UMSARS Item 2. Walking impairment defined by a score of ≥3 on UMSARS Item 7. Occurrence of falls more than once per week, defined by a score of ≥3 on UMSARS Item 8.
Severe hepatic insufficiency: ALT ≥ 2.0 × upper limit of normal (ULN) or AST ≥ 2.0 × ULN.
Severe renal insufficiency: Serum creatinine ≥ 1.5 × ULN or estimated Glomerular Filtration Rate (eGFR) < 40 mL/min/1.73 m².
Severe cardiac insufficiency: New York Heart Association (NYHA) Class 3 or 4.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Meng Cai, Ph.D | Contact | 0086-027-59337986 | caimeng@iregene.com | |
| Jing Zhao, PhD | Contact | zhaojing@iregene.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Beijing Tiantan Hospital, Capital Medical University | Recruiting | Beijing | Beijing Municipality | 100730 | China |
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| 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. | 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. | 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. | 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. | Weeks 13, 26, and 52 post-transplant |
| Weeks 13, 26, and 52 post-transplant |