A Phase I/III Clinical Study to Evaluate NouvNeu001 Injection for Multiple System Atrophy
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age30-70
SponsoriRegene Therapeutics Co., Ltd.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Aged between 30 and 70 years (inclusive), regardless of gender.
The subject understands and agrees to comply with the study procedures and voluntarily provides written informed consent.
Diagnosed with pathologically confirmed, clinically established, or clinically probable Multiple System Atrophy (MSA) according to the 2022 MDS diagnostic criteria.
Current treatments for core MSA symptoms are inadequately controlled.
Disqualifiers
Neurological diseases/disorders other than Multiple System Atrophy, such as Parkinson's disease, Dementia with Lewy Bodies, Essential Tremor, Progressive Supranuclear Palsy, Spinocerebellar Ataxia, Hereditary Spastic Paraplegia, Corticobasal Degeneration, Vascular Parkinsonism, Normal Pressure Hydrocephalus, or Drug-induced/Postencephalitic Parkinsonism.
Diagnosis of dementia.
Previous or current receipt of other disease-modifying therapies, or participation in clinical trials of other new drugs or novel therapies.
Use of medications within the past 3 months that may affect Parkinsonian symptoms, autonomic function, or the evaluation of safety.
Trial design
Design model
Sequential
Treatments tested in this trial
Human Dopaminergic Progenitor Cells
Biological/VaccineSingle injection of Human Dopaminergic Progenitor Cells into the putamen/striatum region of brain.
Treatment groups
9 Participants
are divided into 2 treatment groupsGroup A: NouvNeu001Experimental treatment 1 intervention
Group B: controlNo intervention 0 interventions
Trial outcomes
Primary outcomes
1
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-transplantSecondary outcomes
1
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-transplant2
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-transplant3
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-transplant4
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-transplantOther outcomes
1
Pharmacodynamic Biomarkers (NfL)
measurement of neurofilament light chain (NfL) levels in human blood samples.
Time frame
Weeks 13, 26, and 52 post-transplant2
Pharmacodynamic Biomarkers (α-syn)
measurement of alpha-synuclein (α-syn) levels in human blood samples.
Time frame
Weeks 13, 26, and 52 post-transplantSponsors and contacts
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Source ClinicalTrials.gov