Multiple System Atrophy - Parkinsonian Subtype (MSA-P)Phase 1NCT07289477
A Phase I/III Clinical Study to Evaluate NouvNeu001 Injection for Multiple System Atrophy
A Phase 1 study testing Human Dopaminergic Progenitor Cells in people with Multiple System Atrophy - Parkinsonian Subtype (MSA-P), run by iRegene Therapeutics Co., Ltd. at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 30 to 70 years
- Conditions the study is for
- Multiple System Atrophy - Parkinsonian Subtype (MSA-P)
- What is being tested?
- Human Dopaminergic Progenitor Cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 5 Jan 2026to Jul 2031
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 30 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- The duration of MSA-related motor symptoms (parkinsonism and/or cerebellar ataxia) is no more than 5 years.
- Ability to walk without human assistance, defined as being able to take at least 10 steps; the use of assistive devices (e.g., a walker or cane) is permitted.
- Life expectancy of at least 3 years.
- The subject agrees not to participate in any other clinical studies for 24 months following the investigational product administration.
Who cannot join
- 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.
- Presence of clinically significant or unstable medical or surgical conditions that may preclude the safe completion of the treatment or confound the treatment outcomes.
- History of or undergoing treatment for recurrent stroke.
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- Screening brain MRI shows other significant pathological findings, including but not limited to: cerebral hemorrhage, acute cerebral infarction, aneurysm, vascular malformation, infectious lesions, brain tumors, or other space-occupying lesions (meningiomas or arachnoid cysts with a maximum diameter of less than 1 cm do not warrant exclusion).
- Subjects meeting any of the following criteria indicating advanced disease:
- 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.
- History of current substance abuse and/or alcohol abuse (within 12 months prior to screening).
- Known allergy to the investigational product(s); or history of allergy to antibiotics or other drugs.
- Positive screening results for active viral infection, including Human Immunodeficiency Virus (HIV), Hepatitis B Surface Antigen (HBsAg), Hepatitis B Core Antibodies, or Hepatitis C Virus (HCV).
- Severe hepatic insufficiency, renal insufficiency, or severe cardiac insufficiency:
- 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.
- History of thrombocytopenia within the past three months, other bleeding disorders, or current receipt of anticoagulant therapy (excluding aspirin at a dose ≤ 100 mg per day).
- Pregnancy, lactation, or planning pregnancy.
- History of Bipolar Disorder, Major Depressive Disorder, Schizophrenia, or other psychotic disorders.
- Subjects judged by the investigator to have suicidal ideation at screening, or a suicide attempt within 6 months prior to screening.
- Contraindications for surgery (e.g., implantation of cochlear implants, pacemakers, defibrillators, history of stereotactic ablation, previous implantation of unilateral/bilateral similar products) or history of other surgeries within the past 6 months deemed by the investigator to potentially affect this trial, or other neurosurgical contraindications.
- Clinically significant cardiac disease defined as: myocardial infarction, NYHA Class III or IV heart failure, uncontrolled coronary spasm, severe uncontrolled ventricular arrhythmia, or evidence of acute ischemia or abnormal conduction system on ECG within 6 months prior to enrollment.
- Diagnosis of malignancy.
- Family history of congenital or inherited immunodeficiency disorders.
- Considered by the investigator to have poor compliance.
- Any other significant disease or condition that, in the investigator's judgment, may jeopardize the subject's safety or interfere with the study assessments.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Jing Zhao, PhD contact
China1 site, 1 recruiting
- Recruiting
Beijing Tiantan Hospital, Capital Medical University
Beijing, Beijing Municipality, 100730
Yilong Wang, 010-59978538, yilong528@aliyun.com
About this study
Official title: A Phase I/III Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebral Putamen Transplantation of NouvNeu001 Injection for Multiple System Atrophy
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.
- Being tested
- Human Dopaminergic Progenitor Cells
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human Dopaminergic Progenitor CellsBiological
Single injection of Human Dopaminergic Progenitor Cells into the putamen/striatum region of brain.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: Human Dopaminergic Progenitor Cells |
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of Adverse Events as Assessed by CTCAE V5.0
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs).
Measured: 52 weeks post-transplant
Secondary outcomes (5)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Weeks 13, 26, and 52 post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2025
- Study start5 Jan 2026
- Primary completionOct 2027 (estimated)
- Study completionJul 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple System Atrophy - Parkinsonian Subtype (MSA-P)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- iRegene Therapeutics Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07289477
- Sponsor's protocol ID
- NouvNeu004-01
- Status last verified
- Nov 2025
- First submitted
- 26 Nov 2025
- Last updated
- 14 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/III Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebral Putamen Transplantation of NouvNeu001 Injection for Multiple System Atrophy
- ClinicalTrials.gov ID
- NCT07289477
- Acronym / sponsor ID
- NouvNeu004-01
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- iRegene Therapeutics Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 5 Jan 2026 (actual)
- Primary completion
- Oct 2027 (estimated)
- Study completion
- Jul 2031 (estimated)
- First posted
- 17 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NouvNeu001 | Experimental | Biological: Human Dopaminergic Progenitor CellsDuring 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. |
| 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. |
Who may qualify
- Ages
- 30 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
- The duration of MSA-related motor symptoms (parkinsonism and/or cerebellar ataxia) is no more than 5 years.
Key exclusion criteria (26 listed)
- 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.
- Presence of clinically significant or unstable medical or surgical conditions that may preclude the safe completion of the treatment or confound the treatment outcomes.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Human Dopaminergic Progenitor CellsSingle injection of Human Dopaminergic Progenitor Cells into the putamen/striatum region of brain. | Biological | NouvNeu001 |
Primary outcome
Incidence and severity of Adverse Events as Assessed by CTCAE V5.0
Time frame: 52 weeks post-transplant
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs).
5 secondary outcomes
Change from baseline in the UMSARS total score (Part I + Part II) at Weeks 13, 26, and 52.
Time frame: Weeks 13, 26, and 52 post-transplant
Change from baseline in the UMSARS Part I score at Weeks 13, 26, and 52.
Time frame: Weeks 13, 26, and 52 post-transplant
Change from baseline in the UMSARS Part II score at Weeks 13, 26, and 52.
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.
Time frame: Weeks 52 post-transplant
Change from baseline in the CGI-S score at Weeks 13, 26, and 52.
Time frame: Weeks 13, 26, and 52 post-transplant
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jing Zhao, PhD contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Human Dopaminergic Progenitor Cells in people with Multiple System Atrophy - Parkinsonian Subtype (MSA-P), run by iRegene Therapeutics Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Meng Cai, Ph.D (0086-027-59337986, caimeng@iregene.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of Adverse Events as Assessed by CTCAE V5.0 (time frame: 52 weeks post-transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Apr 2026.